Clinical Trial Results:
A multi-center, open-label trial of the long-term efficacy and safety of Lamazym for the treatment of patients with alpha-Mannosidosis.
Summary
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EudraCT number |
2011-004355-40 |
Trial protocol |
DK DE BE GB ES |
Global end of trial date |
20 Sep 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
11 Aug 2016
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First version publication date |
11 Aug 2016
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
rhLAMAN-04
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01681940 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Zymenex A/S
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Sponsor organisation address |
Roskildevej 12C, Hilleroed, Denmark, 3400
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Public contact |
Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., clinicalTrials_info@chiesi.com
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Scientific contact |
Clinical Trial Transparency, Chiesi Farmaceutici S.p.A., clinicalTrials_info@chiesi.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-001056-PIP02-12 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
20 Sep 2013
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
20 Sep 2013
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Global end of trial reached? |
Yes
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Global end of trial date |
20 Sep 2013
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The overall objective of this trial is the evaluation of long-term efficacy, safety and tolerability of Lamazym treatment in patients with alpha-Mannosidosis.
The primary objectives of the trial are:
Evaluation of long-term efficacy from baseline of Lamazym on reduction of the biomarker Oligosaccharides in blood and CSF and an improvement in the 3 minute stair climb, 6 minute walk test and pulmonary function from baseline.
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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.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
22 Jan 2012
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Denmark: 10
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Worldwide total number of subjects |
10
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EEA total number of subjects |
10
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
5
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Adolescents (12-17 years) |
5
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
A total of 10 patients were enrolled in the rhLAMAN-02 and rhLAMAN-03 trials. One (1) patient (Patient 03) was withdrawn from treatment in the rhLAMAN-03 trial after approximately 2.5 months of treatment and was subsequently withdrawn from the trial. All 9 patients who completed the rhLAMAN-03 trial continued into the rhLAMAN-04 trial. | ||||||||||
Pre-assignment
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Screening details |
No patients failed screening or withdrew from the rhLAMAN-04 trial | ||||||||||
Period 1
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Period 1 title |
overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
Arms
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Arm title
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Lamazym | ||||||||||
Arm description |
In the Laman04 study, all patients were treated with the same dose: 1 mg/kg body weight, and each patient received weekly i.v. infusions of Lamazym with 7 days ± 2 days required between each infusion | ||||||||||
Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Lamazym
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Investigational medicinal product code |
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Other name |
recombinant human alpha-mannosidase
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Pharmaceutical forms |
Powder and solution for solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
In the Laman04 all patients were treated with the same dose: 1 mg/kg body weight, and each patient received weekly i.v. infusions of Lamazym with 7 days ± 2 days required between each infusion
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Baseline characteristics reporting groups
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Reporting group title |
overall trial
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Baseline
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Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
The full analysis set (FAS) includes all 10 patients receiving the investigational medicinal product in at least one of the rhLAMAN-02, rhLAMAN-03 and rhLAMAN-04. The FAS corresponds to the efficacy analysis set.
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Subject analysis set title |
End evaluation
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Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
End evaluation is made at Visit 26a of the rhLAMAN04 study
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End points reporting groups
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Reporting group title |
Lamazym
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Reporting group description |
In the Laman04 study, all patients were treated with the same dose: 1 mg/kg body weight, and each patient received weekly i.v. infusions of Lamazym with 7 days ± 2 days required between each infusion | ||
Subject analysis set title |
Baseline
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
The full analysis set (FAS) includes all 10 patients receiving the investigational medicinal product in at least one of the rhLAMAN-02, rhLAMAN-03 and rhLAMAN-04. The FAS corresponds to the efficacy analysis set.
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Subject analysis set title |
End evaluation
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
End evaluation is made at Visit 26a of the rhLAMAN04 study
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End point title |
Change from baseline in blood serum oligosaccharides | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.1 of the CSR
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End point type |
Primary
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End point timeframe |
Serum oligosaccharides were measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
< 0.001 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in CSF oligosaccharides | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.3 of the CSR
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End point type |
Primary
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End point timeframe |
CSF oligosaccharides were measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.086 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in 3-min stair climb | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.4 of the CSR
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End point type |
Primary
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End point timeframe |
Stair climbing was assessed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.004 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in 6-min walk test | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.5 of the CSR
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End point type |
Primary
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End point timeframe |
6-minute walk test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.009 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in pulmonary FVC | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.6 of the CSR
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End point type |
Primary
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End point timeframe |
FVC was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.003 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline of pulmonary FVC as percent of predicted normal value | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.7 of the CSR
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End point type |
Primary
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End point timeframe |
FVC was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 12), and at rhLAMAN-04 evaluation (Visit 26a, month 18).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.157 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in pulmonary FEV1 | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.8 of the CSR
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End point type |
Primary
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End point timeframe |
FEV1 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 12), and at rhLAMAN-04 evaluation (Visit 26a, month 18).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
End evaluation v Baseline
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.035 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in pulmonary FEV1 as percent of predicted normal value | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.9 of the CSR
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End point type |
Primary
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End point timeframe |
FEV1 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
End evaluation v Baseline
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.985 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in pulmonary peak expiratory flow rate | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.10 of the CSR
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End point type |
Primary
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End point timeframe |
PEF rate was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.23 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in BOT2 balance | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.11 of the CSR
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End point type |
Secondary
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End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 12), and at rhLAMAN-04 evaluation (Visit 26a, month 18).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
End evaluation v Baseline
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.946 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in BOT2 bilateral coordination | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.12 of the CSR
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End point type |
Secondary
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End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 12), and at rhLAMAN-04 evaluation (Visit 26a, month 18).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.007 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in BOT2 running speed and agility | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.13 of the CSR
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End point type |
Secondary
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End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.048 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in BOT2 upper limb coordination | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.14 of the CSR
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End point type |
Secondary
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End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
Baseline v End evaluation
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.254 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in BOT2 manual dexterity | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.15 of the CSR
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End point type |
Secondary
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End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 12), and at rhLAMAN-04 evaluation (Visit 26a, month 18).
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
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Comparison groups |
End evaluation v Baseline
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Number of subjects included in analysis |
19
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
P-value |
= 0.055 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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End point title |
Change from baseline in BOT2 fine motor integration | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.16 of the CSR
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End point type |
Secondary
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End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
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|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.737 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in BOT2 fine motor precision | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.17 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
BOT2 was measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 12), and at rhLAMAN-04 evaluation (Visit 26a, month 18).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.009 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in CSF-GFAp | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.18 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
CSF biomarkers were measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
< 0.001 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in CSF-NFLp | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.19 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
CSF biomarkers were measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.016 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in CSF-Tau | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.20 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
CSF biomarkers were measured at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.159 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Design analogies | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.26 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.137 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Figure ground | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.27 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.372 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Form completion | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.28 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.022 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Paper folding | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.29 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.163 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Repeated pattern | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.30 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.544 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Sequential order | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.31 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.125 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in Leiter R test score - Total equivalence age 1 | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.32 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Leiter R test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.022 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in audiometric best ear bone conduction | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.33 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Hearing test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.277 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in audiometric left ear air conduction | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.34 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Hearing test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.159 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in audiometric right ear air conduction | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.35 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Hearing test was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.057 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in CHAQ - pain on VAS | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.37 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
CHAQ was administered at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.578 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in CHAQ - general evaluation on VAS | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.38 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
CHAQ was administered at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
17
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.673 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRI ADC grey matter | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.39 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRI was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.358 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRI ADC standard | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.40 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRI was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.614 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRI ADC white matter | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.41 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRI was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.139 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRS mannose complex visual grey matter | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.42 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRS was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.081 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRS mannose complex visual standard | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.43 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRS was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 1 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRS mannose complex visual white matter | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.44 of the CSR
|
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End point type |
Secondary
|
||||||||||||
End point timeframe |
MRS was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
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Comparison groups |
Baseline v End evaluation
|
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Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
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Analysis type |
other | ||||||||||||
P-value |
= 0.347 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRS numerical mannose complex index grey matter | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.45 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRS was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
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|
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Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
End evaluation v Baseline
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.54 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRS numerical mannose complex index standard | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
See Table 14.2.1.46 of the CSR
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRS was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
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|
|||||||||||||
Statistical analysis title |
End of LAMAN04 vs Baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.839 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
|
|||||||||||||
End point title |
Change from baseline in MRS numerical mannose complex index white matter | ||||||||||||
End point description |
Baseline data for efficacy endpoints was recorded in the rhLAMAN-02 trial (the last value available before first dosing in rhLAMAN-02 was always used as the baseline value).
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
MRS was performed at baseline (rhLAMAN-02 Visit 2), at interim evaluation (Visit 13a, month 3), and at rhLAMAN-04 evaluation (Visit 26a, month 6).
|
||||||||||||
|
|||||||||||||
Statistical analysis title |
End evaluation vs baseline | ||||||||||||
Statistical analysis description |
End evaluation is made at Visit 26a of the LAMAN04 study
|
||||||||||||
Comparison groups |
Baseline v End evaluation
|
||||||||||||
Number of subjects included in analysis |
19
|
||||||||||||
Analysis specification |
Pre-specified
|
||||||||||||
Analysis type |
other | ||||||||||||
P-value |
= 0.733 | ||||||||||||
Method |
ANCOVA | ||||||||||||
Confidence interval |
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Adverse events information
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Timeframe for reporting adverse events |
AE were recorded at interim evaluation (Visit 13a, month 12), at each dose visit (Visits 2-88), at rhLAMAN-04 evaluation (Visit 26a, month 18) and at Visit 89 (end of trial).
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Adverse event reporting additional description |
The reporting was based on the full analysis set (FAS) and the safety analysis set (the safety analysis set was defined as all patients exposed to at least one dose of IMP) and included all 10 patients who initiated treatment with Lamazym in the rhLAMAN-02 trial, and out of which 9 patients continued into the rhLAMAN-04 trial.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
16.0
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Reporting groups
|
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Reporting group title |
Safety analysis set
|
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Reporting group description |
The safety analysis set was defined as all patients exposed to at least one dose of IMP) and included all 10 patients who initiated treatment with Lamazym in the rhLAMAN-02 trial, and out of which 9 patients continued into the rhLAMAN-04 trial | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 1% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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|
|||
Substantial protocol amendments (globally) |
|||
Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
||
21 Aug 2012 |
The period subjects will receive one dose of enzyme every week passes from 6 months to 10 months.
- Continuation of the treatment passes from a total of 30 ± 4 weeks to a total of 40 ± 6 weeks. So that the EOT is numbered Visit #47 instead of Visit #35
- The dose visits becomes numbered 2-46 instead of 2-34.
|
||
13 Nov 2012 |
- The period subjects will receive one dose of enzyme every week passes from 6 months to 16 months.
- Continuation of the treatment passes from a total of 30 ± 4 weeks to a total of 72 weeks. So that the EOT is numbered Visit #73 instead of Visit #35
- The dose visits becomes numbered 2-72 instead of 2-34.
|
||
16 Apr 2013 |
The period subjects will receive one dose of enzyme every week passes from 6 months to 20 months.
- Continuation of the treatment passes from a total of 30 ± 4 weeks to a total of 88 weeks. So that the EOT is numbered Visit #89 instead of Visit #35
- The dose visits becomes numbered 2-89 instead of 2-34.
|
||
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. | |||
No caveats or limitations are applicable to this summary of the results |