Clinical Trial Results:
A single center, open label clinical trial investigating the longterm efficacy of rhLAMAN (recombinant human alphamannosidase or Lamazym) treatment in subjects with alphaMannosidosis who previously participated in Lamazym trials.
Summary


EudraCT number 
201400395015 
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 
rhLAMAN10


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 longterm efficacy of Lamazym treatment in patients with alphamannosidosis 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 longterm treatment with Lamazym in patients with alphamannosidosis 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 longterm data collection, the data collected during the present study was to be integrated with that from four previous studies (rhLAMAN02, rhLAMAN03, rhLAMAN04 and rhLAMAN05) and two ongoing studies (rhLAMAN07 and rhLAMAN09).


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 followup 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 (027 days) 
0


Infants and toddlers (28 days23 months) 
0


Children (211 years) 
9


Adolescents (1217 years) 
10


Adults (1864 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 LAMAN02/03 and later in LAMAN05 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.  
Preassignment


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 openlabel 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 alphamannosidase


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 postbaseline. The endpoint 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 
Subgroup analysis  
Subject analysis set description 
Baseline values were defined as the last nonmissing value before the first dose of Lamazym (derived from parental trials rhLAMAN02 and rhLAMAN05, respectively).


Subject analysis set title 
Last observation population


Subject analysis set type 
Subgroup 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 postbaseline. The endpoint 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 
Subgroup analysis  
Subject analysis set description 
Baseline values were defined as the last nonmissing value before the first dose of Lamazym (derived from parental trials rhLAMAN02 and rhLAMAN05, respectively).


Subject analysis set title 
Last observation population


Subject analysis set type 
Subgroup 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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.004  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.05  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.071  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.007  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.011  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.226  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.241  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.002  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.003  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.004  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
10.912  
upper limit 
53.577 


End point title 
Absolute change from baseline to last observation in BOT2  
End point description 
BOT2 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: BOT2.pdf) 

Statistical analysis title 
Last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.23  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
3.4  
upper limit 
13.6 


End point title 
% change from baseline to last observation in BOT2  
End point description 
BOT2 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: BOT2.pdf) 

Statistical analysis title 
last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.035  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.094  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.336  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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: CSFTau.pdf) 

Statistical analysis title 
last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.468  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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: CSFTau.pdf) 

Statistical analysis title 
Last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.106  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
1.7  
upper limit 
17.2 


End point title 
Absolute change from baseline to last observation in CSFNFL  
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: CSFNFL.pdf) 

Statistical analysis title 
Last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.477  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
100  
upper limit 
48 


End point title 
% change from baseline to last observation in CSFNFL  
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: CSFNFL.pdf) 

Statistical analysis title 
last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.158  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
4.8  
upper limit 
28 


End point title 
Absolute change from baseline to last observation in CSFGFAP  
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: CSFGFAP.pdf) 

Statistical analysis title 
last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.053  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
2  
upper limit 
351.7 


End point title 
% change from baseline to last observation in CSFGFAP  
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: CSFGFAP.pdf) 

Statistical analysis title 
last observation vs baseline  
Statistical analysis description 
In a singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.023  
Method 
paired 2sided T test  
Confidence interval 

sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.006  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.619  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.172  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.674  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.782  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.03  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.05  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.438  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.878  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.095  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.768  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.139  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.485  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.543  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.471  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
18.8  
upper limit 
39.48 


End point title 
Absolute change from baseline to last observation in EQ5D5L 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: EQ5DL.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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.08  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


lower limit 
0.0066  
upper limit 
0.1075 


End point title 
% change from baseline to last observation in EQ5D5L 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: EQ5DL.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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.0036  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
< 0.001  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.699  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.83  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.007  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.003  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.035  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.05  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.598  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.611  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 1  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.815  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.329  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.705  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.704  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.504  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.287  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 singlearm 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 
Prespecified


Analysis type 
other  
Pvalue 
= 0.813  
Method 
paired 2sided T test  
Confidence interval 

level 
95%  
sides 
2sided


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 populationsafety


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 nonserious 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 