Clinical Trial Results:
A single center, open label clinical trial investigating the long-term efficacy of rhLAMAN (recombinant human alpha-mannosidase or Lamazym) treatment in subjects with alpha-Mannosidosis who previously participated in Lamazym trials.
Summary
|
|
EudraCT number |
2014-003950-15 |
Trial protocol |
DK |
Global end of trial date |
13 Jun 2015
|
Results information
|
|
Results version number |
v1(current) |
This version publication date |
29 Jul 2016
|
First version publication date |
29 Jul 2016
|
Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
|
|||
Trial identification
|
|||
Sponsor protocol code |
rhLAMAN-10
|
||
Additional study identifiers
|
|||
ISRCTN number |
- | ||
US NCT number |
NCT02478840 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
|
|||
Sponsor organisation name |
Zymenex A/S
|
||
Sponsor organisation address |
Roskildevej 12C, Hilleroed, Denmark, 3400
|
||
Public contact |
Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., +39 0521 2791, clinicalTrials_info@chiesi.com
|
||
Scientific contact |
Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., +39 0521 2791, clinicaltrials_info@chiesi.com
|
||
Paediatric regulatory details
|
|||
Is trial part of an agreed paediatric investigation plan (PIP) |
No
|
||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Results analysis stage
|
|||
Analysis stage |
Final
|
||
Date of interim/final analysis |
13 Jun 2015
|
||
Is this the analysis of the primary completion data? |
Yes
|
||
Primary completion date |
13 Jun 2015
|
||
Global end of trial reached? |
Yes
|
||
Global end of trial date |
13 Jun 2015
|
||
Was the trial ended prematurely? |
No
|
||
General information about the trial
|
|||
Main objective of the trial |
The overall objective of the trial was the evaluation of the long-term efficacy of Lamazym treatment in patients with alpha-mannosidosis who were previously enrolled in trials with Lamazym and were currently receiving the treatment according to the AfterCare Program agreed with the National Authorities.
Primary objective: The primary objective of the trial was to evaluate the impact of the long-term treatment with Lamazym in patients with alpha-mannosidosis upon the level of biomarker oligosaccharides in serum and upon the endurance as measured by the change from Baseline in the number of steps climbed in 3 minutes (three minute stair climb test, 3MSCT).
To address the need for long-term data collection, the data collected during the present study was to be integrated with that from four previous studies (rhLAMAN-02, rhLAMAN-03, rhLAMAN-04 and rhLAMAN-05) and two ongoing studies (rhLAMAN-07 and rhLAMAN-09).
|
||
Protection of trial subjects |
The study was conducted in accordance with the Declaration of Helsinki, Good Clinical Practice (GCP) guidelines and local law requirements. Other than routine care, no specific measures for protection of
trial subjects were implemented.
|
||
Background therapy |
There was no restriction on the use of concomitant medications/therapies during the trial. There are currently no known interactions of Lamazym with other medications. | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
16 Feb 2015
|
||
Long term follow-up planned |
No
|
||
Independent data monitoring committee (IDMC) involvement? |
No
|
||
Population of trial subjects
|
|||
Number of subjects enrolled per country |
|||
Country: Number of subjects enrolled |
Denmark: 33
|
||
Worldwide total number of subjects |
33
|
||
EEA total number of subjects |
33
|
||
Number of subjects enrolled per age group |
|||
In utero |
0
|
||
Preterm newborn - gestational age < 37 wk |
0
|
||
Newborns (0-27 days) |
0
|
||
Infants and toddlers (28 days-23 months) |
0
|
||
Children (2-11 years) |
9
|
||
Adolescents (12-17 years) |
10
|
||
Adults (18-64 years) |
14
|
||
From 65 to 84 years |
0
|
||
85 years and over |
0
|
|
|||||||
Recruitment
|
|||||||
Recruitment details |
A total of 34 patients (pts) were recruited with 35 pts identifiers; 1 patient participated in LAMAN-02/03 and later in LAMAN-05 and was counted once. Of the 34 pts, one patient received placebo in LAMAN 05 and then Lamazym in the AfterCare program. This patient discontinued shortly after starting the program and was excluded from all analyses. | ||||||
Pre-assignment
|
|||||||
Screening details |
Of the 34 patients recruited, 1 (from the LAMAN05 study) discontinued the treatment shortly after starting the AfterCare program. As such, no data for this patient was collected during the active treatment, and so this patient was excluded from all analyses. Hence, 33 patients in total were actually analysed. | ||||||
Period 1
|
|||||||
Period 1 title |
Overall trial (overall period)
|
||||||
Is this the baseline period? |
Yes | ||||||
Allocation method |
Not applicable
|
||||||
Blinding used |
Not blinded | ||||||
Blinding implementation details |
This was an open-label study.
|
||||||
Arms
|
|||||||
Arm title
|
Lamazym 1mg/kg body weight | ||||||
Arm description |
Data displays were generally for patients overall. Each patient received the weekly dose of Lamazym of 1mg/kg. | ||||||
Arm type |
Experimental | ||||||
Investigational medicinal product name |
Lamazym
|
||||||
Investigational medicinal product code |
|||||||
Other name |
recombinant human lysosomal alpha-mannosidase
|
||||||
Pharmaceutical forms |
Solution for infusion
|
||||||
Routes of administration |
Intravascular use
|
||||||
Dosage and administration details |
Lamazym, 1mg/kg body weight as intravenous infusion. Each patient received the weekly dose of Lamazym (as scheduled by their AfterCare program) in connection with an evaluation visit.
|
||||||
|
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Baseline characteristics reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Lamazym 1mg/kg body weight
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Data displays were generally for patients overall. Each patient received the weekly dose of Lamazym of 1mg/kg. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis sets
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set title |
Overall - FAS
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Full Analysis Set (FAS): consisted of all patients who were dosed and whose efficacy was evaluated post-baseline. The end-point values for this Subject Analysis set reported are the changes from baseline to last observation.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set title |
Overall - safety population
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set type |
Safety analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
The safety analysis set (SAS) consisted of all patients who received at least one dose of trial medication.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set title |
Baseline population
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set type |
Sub-group analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Baseline values were defined as the last non-missing value before the first dose of Lamazym (derived from parental trials rhLAMAN-02 and rhLAMAN-05, respectively).
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set title |
Last observation population
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set type |
Sub-group analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Last observation values were defined as the last available value at the end of rhLAMAN trials (derived from the last trial the patient participated in).
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|
|||
End points reporting groups
|
|||
Reporting group title |
Lamazym 1mg/kg body weight
|
||
Reporting group description |
Data displays were generally for patients overall. Each patient received the weekly dose of Lamazym of 1mg/kg. | ||
Subject analysis set title |
Overall - FAS
|
||
Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Full Analysis Set (FAS): consisted of all patients who were dosed and whose efficacy was evaluated post-baseline. The end-point values for this Subject Analysis set reported are the changes from baseline to last observation.
|
||
Subject analysis set title |
Overall - safety population
|
||
Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
The safety analysis set (SAS) consisted of all patients who received at least one dose of trial medication.
|
||
Subject analysis set title |
Baseline population
|
||
Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Baseline values were defined as the last non-missing value before the first dose of Lamazym (derived from parental trials rhLAMAN-02 and rhLAMAN-05, respectively).
|
||
Subject analysis set title |
Last observation population
|
||
Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Last observation values were defined as the last available value at the end of rhLAMAN trials (derived from the last trial the patient participated in).
|
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in serum oligosaccharides | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Primary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation.
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: SERUM OLIGOSACCARIDES.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-5.74 | ||||||||||||||||
upper limit |
-3.45 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in serum oligosaccharides | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Primary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: SERUM OLIGOSACCARIDES.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-74.7 | ||||||||||||||||
upper limit |
-50.8 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in the 3MSCT | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Primary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: 3MSCT.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
2.645 | ||||||||||||||||
upper limit |
10.12 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in the 3MSCT | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Primary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: 3MSCT.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.004 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
4.609 | ||||||||||||||||
upper limit |
22.92 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in the 6MSCT | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: 6MWT.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.05 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0 | ||||||||||||||||
upper limit |
44.8 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in the 6MSCT | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: 6MWT.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.071 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0.7 | ||||||||||||||||
upper limit |
14.9 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in FVC (%) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FVC %.pdf) |
||||||||||||||||
Notes [1] - This is the actual number of subjects on which the analysis was made [2] - This is the actual number of subjects on which the analysis was made [3] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.007 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
2.4 | ||||||||||||||||
upper limit |
13.7 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in FVC (%) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FVC %.pdf) |
||||||||||||||||
Notes [4] - This is the actual number of subjects on which the analysis was made [5] - This is the actual number of subjects on which the analysis was made [6] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.011 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
2.6 | ||||||||||||||||
upper limit |
18.5 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in FVC (L) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FVC L.pdf) |
||||||||||||||||
Notes [7] - This is the actual number of subjects on which the analysis was made [8] - This is the actual number of subjects on which the analysis was made [9] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0.31 | ||||||||||||||||
upper limit |
0.845 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in FVC (L) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FVC L.pdf) |
||||||||||||||||
Notes [10] - This is the actual number of subjects on which the analysis was made [11] - This is the actual number of subjects on which the analysis was made [12] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
14.04 | ||||||||||||||||
upper limit |
42.76 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in predicted FEV1 | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FEV %.pdf) |
||||||||||||||||
Notes [13] - This is the actual number of subjects on which the analysis was made [14] - This is the actual number of subjects on which the analysis was made [15] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.226 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-2.4 | ||||||||||||||||
upper limit |
9.9 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in predicted FEV1 | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FEV %.pdf) |
||||||||||||||||
Notes [16] - This is the actual number of subjects on which the analysis was made [17] - This is the actual number of subjects on which the analysis was made [18] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.241 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-3.6 | ||||||||||||||||
upper limit |
13.8 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in FEV1 | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FEV1 L.pdf) |
||||||||||||||||
Notes [19] - This is the actual number of subjects on which the analysis was made [20] - This is the actual number of subjects on which the analysis was made [21] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.002 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0.165 | ||||||||||||||||
upper limit |
0.633 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in FEV1 | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: FEV1 L.pdf) |
||||||||||||||||
Notes [22] - This is the actual number of subjects on which the analysis was made [23] - This is the actual number of subjects on which the analysis was made [24] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.003 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
7.724 | ||||||||||||||||
upper limit |
34.889 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in PEF | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: PEF.pdf) |
||||||||||||||||
Notes [25] - This is the actual number of subjects on which the analysis was made [26] - This is the actual number of subjects on which the analysis was made [27] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0.501 | ||||||||||||||||
upper limit |
1.819 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in PEF | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: PEF.pdf) |
||||||||||||||||
Notes [28] - This is the actual number of subjects on which the analysis was made [29] - This is the actual number of subjects on which the analysis was made [30] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=60 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
60
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.004 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
10.912 | ||||||||||||||||
upper limit |
53.577 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in BOT-2 | ||||||||||||||||
End point description |
BOT-2 total score is expressed as "point score".
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: BOT-2.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.23 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-3.4 | ||||||||||||||||
upper limit |
13.6 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in BOT-2 | ||||||||||||||||
End point description |
BOT-2 total score is expressed as "point score".
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: BOT-2.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.035 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
1 | ||||||||||||||||
upper limit |
25 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in CSF oligosaccharides | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF_OLIGOSACCHARIDES.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.094 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-1.29 | ||||||||||||||||
upper limit |
0.11 |
|
|||||||||||||||||
End point title |
% from baseline to last observation in CSF oligosaccharides | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation.
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF_OLIGOSACCHARIDES.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.336 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-10.9 | ||||||||||||||||
upper limit |
3.84 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in TAU protein | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF-Tau.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.468 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-36.9 | ||||||||||||||||
upper limit |
78.4 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in TAU protein | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF-Tau.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.106 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-1.7 | ||||||||||||||||
upper limit |
17.2 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in CSF-NFL | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF-NFL.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.477 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-100 | ||||||||||||||||
upper limit |
48 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in CSF-NFL | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF-NFL.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.158 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-4.8 | ||||||||||||||||
upper limit |
28 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in CSF-GFA-P | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF-GFA-P.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.053 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-2 | ||||||||||||||||
upper limit |
351.7 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in CSF-GFA-P | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CSF-GFA-P.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
49.6 | ||||||||||||||||
upper limit |
154.3 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in serum immunoglobulin G | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: IGg.pdf) |
||||||||||||||||
Notes [31] - This is the actual number of subjects on which the analysis was made [32] - This is the actual number of subjects on which the analysis was made [33] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
48
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
2.39 | ||||||||||||||||
upper limit |
3.71 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in serum Immunoglobulin G | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: IGg.pdf) |
||||||||||||||||
Notes [34] - This is the actual number of subjects on which the analysis [35] - This is the actual number of subjects on which the analysis [36] - This is the actual number of subjects on which the analysis |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
48
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
32.58 | ||||||||||||||||
upper limit |
55.57 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in Leiter R Total Equivalent Age VR | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Leiter Scale VR.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.023 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0.039 | ||||||||||||||||
upper limit |
0.491 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in Leiter R Total Equivalent Age VR | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Leiter Scale VR.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.006 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
1.633 | ||||||||||||||||
upper limit |
9.043 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in Leiter R Total Equivalent Age AM | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Leiter Scale AM.pdf) |
||||||||||||||||
Notes [37] - This is the actual number of subjects on which the analysis was made [38] - This is the actual number of subjects on which the analysis was made [39] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
48
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.619 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.485 | ||||||||||||||||
upper limit |
0.798 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in Leiter R Total Equivalent Age AM | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Leiter Scale AM.pdf) |
||||||||||||||||
Notes [40] - This is the actual number of subjects on which the analysis was made [41] - This is the actual number of subjects on which the analysis was made [42] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
48
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.172 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-4.372 | ||||||||||||||||
upper limit |
23.063 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in bone conduction (best ear) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Bone conduction best ear.pdf) |
||||||||||||||||
Notes [43] - This is the actual number of subjects on which the analysis was made [44] - This is the actual number of subjects on which the analysis was made [45] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.674 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-2.86 | ||||||||||||||||
upper limit |
1.88 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in bone conduction (best ear) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Bone conduction best ear.pdf) |
||||||||||||||||
Notes [46] - This is the actual number of subjects on which the analysis was made [47] - This is the actual number of subjects on which the analysis was made [48] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.782 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-5.96 | ||||||||||||||||
upper limit |
4.52 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in bone conduction (left ear) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Air conduction left ear.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.03 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-5.36 | ||||||||||||||||
upper limit |
-0.29 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in bone conduction (left ear) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Air conduction left ear.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.05 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-7.58 | ||||||||||||||||
upper limit |
0 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in bone conduction (right ear) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Air conduction right ear.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.438 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-5.06 | ||||||||||||||||
upper limit |
2.25 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in bone conduction (right ear) | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Air conduction right ear.pdf) |
||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.878 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-6.62 | ||||||||||||||||
upper limit |
7.7 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in CHAQ Disability Index | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CHAQ Disability Index.pdf) |
||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.095 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.29 | ||||||||||||||||
upper limit |
0.02 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in CHAQ Disability Index | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CHAQ Disability Index.pdf) |
||||||||||||||||
Notes [49] - This is the actual number of subjects on which the analysis was made [50] - This is the actual number of subjects on which the analysis was made [51] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=66 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
66
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.768 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-18.9 | ||||||||||||||||
upper limit |
14.11 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in CHAQ VAS pain | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CHAQ VAS PAIN.pdf) |
||||||||||||||||
Notes [52] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.139 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.407 | ||||||||||||||||
upper limit |
0.06 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in CHAQ VAS pain | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CHAQ VAS PAIN.pdf) |
||||||||||||||||
Notes [53] - This is the actual number of subjects on which the analysis was made [54] - This is the actual number of subjects on which the analysis was made [55] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.485 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-67 | ||||||||||||||||
upper limit |
32.94 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in CHAQ VAS general | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CHAQ General VAS.pdf) |
||||||||||||||||
Notes [56] - This is the actual number of subjects on which the analysis was made [57] - This is the actual number of subjects on which the analysis was made [58] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.543 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.291 | ||||||||||||||||
upper limit |
0.156 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in CHAQ VAS general | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: CHAQ General VAS.pdf) |
||||||||||||||||
Notes [59] - This is the actual number of subjects on which the analysis was made [60] - This is the actual number of subjects on which the analysis was made [61] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=65 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
65
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.471 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-18.8 | ||||||||||||||||
upper limit |
39.48 |
|
|||||||||||||||||
End point title |
Absolute change from baseline to last observation in EQ-5D-5L Health Index | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: EQ-5D-L.pdf) |
||||||||||||||||
Notes [62] - This is the actual number of subjects on which the analysis was made [63] - This is the actual number of subjects on which the analysis was made [64] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
Last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
48
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.08 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.0066 | ||||||||||||||||
upper limit |
0.1075 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in EQ-5D-5L Health Index | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: EQ-5D-L.pdf) |
||||||||||||||||
Notes [65] - This is the actual number of subjects on which the analysis was made [66] - This is the actual number of subjects on which the analysis was made [67] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=48 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
48
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.0036 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
0.79 | ||||||||||||||||
upper limit |
21.6682 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRI ADC grey | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC Grey.pdf) |
||||||||||||||||
Notes [68] - This is the actual number of subjects on which the analysis was made [69] - This is the actual number of subjects on which the analysis was made [70] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
107.7 | ||||||||||||||||
upper limit |
212.8 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRI ADC grey | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC Grey.pdf) |
||||||||||||||||
Notes [71] - This is the actual number of subjects on which the analysis was made [72] - This is the actual number of subjects on which the analysis was made [73] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
< 0.001 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
13.5 | ||||||||||||||||
upper limit |
27 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRI ADC standard | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC Standard.pdf) |
||||||||||||||||
Notes [74] - This is the actual number of subjects on which the analysis was made [75] - This is the actual number of subjects on which the analysis was made [76] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.699 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-38.3 | ||||||||||||||||
upper limit |
27 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRI ADC standard | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC Standard.pdf) |
||||||||||||||||
Notes [77] - This is the actual number of subjects on which the analysis was made [78] - This is the actual number of subjects on which the analysis was made [79] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.83 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-3.8 | ||||||||||||||||
upper limit |
3.2 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRI ADC white | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC White.pdf) |
||||||||||||||||
Notes [80] - This is the actual number of subjects on which the analysis was made [81] - This is the actual number of subjects on which the analysis was made [82] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.007 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-185 | ||||||||||||||||
upper limit |
-40.8 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRI ADC white | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC White.pdf) |
||||||||||||||||
Notes [83] - This is the actual number of subjects on which the analysis was made [84] - This is the actual number of subjects on which the analysis was made [85] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.003 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-17.8 | ||||||||||||||||
upper limit |
-5.1 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRS mannose complex visual Grey | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Mannose Complex Visual Grey.pdf) |
||||||||||||||||
Notes [86] - This is the actual number of subjects on which the analysis was made [87] - This is the actual number of subjects on which the analysis was made [88] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.035 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.8 | ||||||||||||||||
upper limit |
0 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRS mannose complex visual Grey | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Mannose Complex Visual Grey.pdf) |
||||||||||||||||
Notes [89] - This is the actual number of subjects on which the analysis was made [90] - This is the actual number of subjects on which the analysis was made [91] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.05 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-66.6 | ||||||||||||||||
upper limit |
0 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRS mannose complex visual Standard | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index Standard.pdf) |
||||||||||||||||
Notes [92] - This is the actual number of subjects on which the analysis was made [93] - This is the actual number of subjects on which the analysis was made [94] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.598 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.7 | ||||||||||||||||
upper limit |
0.4 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRS mannose complex visual Standard | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index Standard.pdf) |
||||||||||||||||
Notes [95] - This is the actual number of subjects on which the analysis was made [96] - This is the actual number of subjects on which the analysis was made [97] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.611 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-47.8 | ||||||||||||||||
upper limit |
31.2 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRS mannose complex visual White | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC White.pdf) |
||||||||||||||||
Notes [98] - This is the actual number of subjects on which the analysis was made [99] - This is the actual number of subjects on which the analysis was made [100] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Last observation population v Baseline population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 1 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.7 | ||||||||||||||||
upper limit |
0.7 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRS mannose complex visual White | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: MRI ADC White.pdf) |
||||||||||||||||
Notes [101] - This is the actual number of subjects on which the analysis was made [102] - This is the actual number of subjects on which the analysis was made [103] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Last observation population v Baseline population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.815 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-52.4 | ||||||||||||||||
upper limit |
42.9 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRS numerical mannose complex index grey | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index. Grey.pdf) |
||||||||||||||||
Notes [104] - This is the actual number of subjects on which the analysis was made [105] - This is the actual number of subjects on which the analysis was made [106] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.329 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.5 | ||||||||||||||||
upper limit |
0.2 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRS numerical mannose complex index grey | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index. Grey.pdf) |
||||||||||||||||
Notes [107] - This is the actual number of subjects on which the analysis was made [108] - This is the actual number of subjects on which the analysis was made [109] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=18 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
18
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.705 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-30.7 | ||||||||||||||||
upper limit |
21.7 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRS numerical mannose complex index standard | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index Standard.pdf) |
||||||||||||||||
Notes [110] - This is the actual number of subjects on which the analysis was made [111] - This is the actual number of subjects on which the analysis was made [112] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=16 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
16
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.704 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.4 | ||||||||||||||||
upper limit |
0.3 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRS numerical mannose complex index standard | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index Standard.pdf) |
||||||||||||||||
Notes [113] - This is the actual number of subjects on which the analysis was made [114] - This is the actual number of subjects on which the analysis was made [115] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=16 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
16
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.504 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-34.2 | ||||||||||||||||
upper limit |
18.8 |
|
|||||||||||||||||
End point title |
absolute change from baseline to last observation in MRS numerical mannose complex index white | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index White.pdf) |
||||||||||||||||
Notes [116] - This is the actual number of subjects on which the analysis was made [117] - This is the actual number of subjects on which the analysis was made [118] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.287 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-0.4 | ||||||||||||||||
upper limit |
0.1 |
|
|||||||||||||||||
End point title |
% change from baseline to last observation in MRS numerical mannose complex index white | ||||||||||||||||
End point description |
Only (absolute and percent) changes from baseline to last observation are inserted in the system. For the complete data regarding the other Timepoints see the pdf attached.
|
||||||||||||||||
End point type |
Secondary
|
||||||||||||||||
End point timeframe |
Timepoints: Month 6, Month 12, Month 18, Month 24, Month 36, Month 48 and Last Observation
|
||||||||||||||||
|
|||||||||||||||||
Attachments |
Untitled (Filename: Numerical Mannose Complex Index White.pdf) |
||||||||||||||||
Notes [119] - This is the actual number of subjects on which the analysis was made [120] - This is the actual number of subjects on which the analysis was made [121] - This is the actual number of subjects on which the analysis was made |
|||||||||||||||||
Statistical analysis title |
last observation vs baseline | ||||||||||||||||
Statistical analysis description |
In a single-arm study, groups examined should not be added. The number N=17 (subject analysis set) is an innate error of the EudraCT database system.
|
||||||||||||||||
Comparison groups |
Baseline population v Last observation population
|
||||||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||||||
Analysis type |
other | ||||||||||||||||
P-value |
= 0.813 | ||||||||||||||||
Method |
paired 2-sided T test | ||||||||||||||||
Confidence interval |
|||||||||||||||||
level |
95% | ||||||||||||||||
sides |
2-sided
|
||||||||||||||||
lower limit |
-31.1 | ||||||||||||||||
upper limit |
25.3 |
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
From the first dose to last observation.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Adverse event reporting additional description |
A total of 546 TEAEs were reported in 29 patients. Approxi. one third of patients (12 patients) experienced
serious TEAEs. Only 3 patients experienced TEAEs that were severe in intensity. There were no deaths, and no TEAEs led to treatment
discontinuation.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
16.0
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Overall population-safety
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
The safety analysis set (SAS) consisted of all patients who received at least one dose of trial medication. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Frequency threshold for reporting non-serious adverse events: 1% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|
|||
Substantial protocol amendments (globally) |
|||
Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
|||
Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
There are no limitations or caveats to this summary of results |