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Trial record 1 of 1 for:    LTS12869
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Growth and Development Study of Alglucosidase Alfa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00486889
Recruitment Status : Completed
First Posted : June 15, 2007
Results First Posted : August 26, 2022
Last Update Posted : August 26, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pompe Disease
Glycogen Storage Disease Type II (GSD-II)
Acid Maltase Deficiency Disease
Intervention Biological: alglucosidase alfa
Enrollment 12
Recruitment Details The study was conducted at 3 active sites in United States. A total of 12 participants were screened from 26-August-2008 to 07-February-2014.
Pre-assignment Details All 12 participants were enrolled and treated in the study.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Participants received alglucosidase alfa 20 milligrams per kilogram (mg/kg) body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Period Title: Overall Study
Started 12
Completed 1
Not Completed 11
Reason Not Completed
Death             3
Non-compliant             2
Withdrawal by Subject             6
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
Analysis was performed on full analysis set (FAS) that included all enrolled participants who had received at least one infusion (complete or partial) of alglucosidase alfa.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Months
Number Analyzed 12 participants
11.558  (6.226)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
7
  58.3%
Male
5
  41.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
5
  41.7%
White
7
  58.3%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Recumbent Height/Length of Participants at Baseline  
Mean (Standard Deviation)
Unit of measure:  Centimeter (cm)
Number Analyzed 12 participants
72.95  (10.24)
Body Weight of Participants at Baseline  
Mean (Standard Deviation)
Unit of measure:  Kilogram (Kg)
Number Analyzed 12 participants
8.42  (2.61)
Head Circumference of Participants at Baseline  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 12 participants
45.20  (3.61)
1.Primary Outcome
Title Recumbent Height/Length of Participants in Centimeters (cm)
Hide Description Height/Length of Participants was measured in centimeters. Week is denoted as Wk in time frame section.
Time Frame Participants 1-12:Baseline, Participant1: Wk 52, Participant2: Wk82, Participants 3-4: Wk208, Participant5: Wk12, Participant6: Wk365, Participant7: Wk64, Participant8:Wk156, Participant9:Wk364, Participant10:Wk52, Participant11:Wk156, Participant12:Wk520
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: cm
Participant-1 Baseline Number Analyzed 1 participants
80.4
Participant-1 Week 52 Number Analyzed 1 participants
93.8
Participant-2 Baseline Number Analyzed 1 participants
67.7
Participant-2 Week 82 Number Analyzed 1 participants
91.1
Participant-3 Baseline Number Analyzed 1 participants
69.1
Participant-3 Week 208 Number Analyzed 1 participants
107.5
Participant-4 Baseline Number Analyzed 1 participants
67.0
Participant-4 Week 208 Number Analyzed 1 participants
110.0
Participant-5 Baseline Number Analyzed 1 participants
71.4
Participant-5 Week 12 Number Analyzed 1 participants
71.1
Participant-6 Baseline Number Analyzed 1 participants
83.5
Participant-6 Week 365 Number Analyzed 1 participants
131.0
Participant-7 Baseline Number Analyzed 1 participants
57.2
Participant-7 Week 64 Number Analyzed 1 participants
80.5
Participant-8 Baseline Number Analyzed 1 participants
70.4
Participant-8 Week 156 Number Analyzed 1 participants
110.3
Participant-9 Baseline Number Analyzed 1 participants
61.0
Participant-9 Week 364 Number Analyzed 1 participants
117.0
Participant-10 Baseline Number Analyzed 1 participants
76.7
Participant-10 Week 52 Number Analyzed 1 participants
83.8
Participant-11 Baseline Number Analyzed 1 participants
95.0
Participant-11 Week 156 Number Analyzed 1 participants
115.0
Participant-12 Baseline Number Analyzed 1 participants
76.0
Participant-12 Week 520 Number Analyzed 1 participants
139.7
2.Primary Outcome
Title Body Weight of Participants in Kilograms (kg)
Hide Description Body Weight of Participants was measured in Kilograms (kg). Week is denoted as Wk in time frame section.
Time Frame Participant1-12:Baseline, Participant1:Wk 52, Participant2:Wk82, Participant3: Wk208,Participant4:Wk364,Participant5:Wk12,Participant6:Wk365,Participant7:Wk64,Participant8:Wk156,Participant9:Wk364,Participant10:Wk52,Participant11:Wk156,Participant12:Wk520
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: kilograms
Participant-1 Baseline Number Analyzed 1 participants
10.0
Participant-1 Week 52 Number Analyzed 1 participants
13.8
Participant-2 Baseline Number Analyzed 1 participants
8.0
Participant-2 Week 82 Number Analyzed 1 participants
13.1
Participant-3 Baseline Number Analyzed 1 participants
6.5
Participant-3 Week 208 Number Analyzed 1 participants
17.4
Participant-4 Baseline Number Analyzed 1 participants
6.2
Participant-4 Week 364 Number Analyzed 1 participants
33.3
Participant-5 Baseline Number Analyzed 1 participants
6.5
Participant-5 Week 12 Number Analyzed 1 participants
6.7
Participant-6 Baseline Number Analyzed 1 participants
12.8
Participant-6 Week 365 Number Analyzed 1 participants
47.5
Participant-7 Baseline Number Analyzed 1 participants
5.0
Participant-7 Week 64 Number Analyzed 1 participants
12.2
Participant-8 Baseline Number Analyzed 1 participants
8.9
Participant-8 Week 156 Number Analyzed 1 participants
20.3
Participant-9 Baseline Number Analyzed 1 participants
5.2
Participant-9 Week 364 Number Analyzed 1 participants
23.1
Participant-10 Baseline Number Analyzed 1 participants
10.5
Participant-10 Week 52 Number Analyzed 1 participants
12.8
Participant-11 Baseline Number Analyzed 1 participants
12.1
Participant-11 Week 156 Number Analyzed 1 participants
35.0
Participant-12 Baseline Number Analyzed 1 participants
9.3
Participant-12 Week 520 Number Analyzed 1 participants
46.3
3.Primary Outcome
Title Head Circumference of Participants in Centimeters (cm)
Hide Description Head Circumference of Participants was measured in Centimeters.
Time Frame Participants1-12:Baseline, Participant1:Week(Wk)52, Participant2:Wk82, Participants3and4:Wk208, Participant5:Wk12, Participant6:Wk365, Participant7:Wk64, Participant8:Wk156, Participant9:Wk312, Participant10:Wk52, Participant11:Wk156, Participant12:Wk468
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: cm
Participant-1 Baseline Number Analyzed 1 participants
45.3
Participant-1 Week 52 Number Analyzed 1 participants
48.1
Participant-2 Baseline Number Analyzed 1 participants
44.0
Participant-2 Week 82 Number Analyzed 1 participants
49.3
Participant-3 Baseline Number Analyzed 1 participants
43.4
Participant-3 Week 208 Number Analyzed 1 participants
52.8
Participant-4 Baseline Number Analyzed 1 participants
42.6
Participant-4 Week 208 Number Analyzed 1 participants
52.4
Participant-5 Baseline Number Analyzed 1 participants
44.3
Participant-5 Week 12 Number Analyzed 1 participants
44.5
Participant-6 Baseline Number Analyzed 1 participants
49.9
Participant-6 Week 365 Number Analyzed 1 participants
56.3
Participant-7 Baseline Number Analyzed 1 participants
39.6
Participant-7 Week 64 Number Analyzed 1 participants
48.0
Participant-8 Baseline Number Analyzed 1 participants
50.8
Participant-8 Week 156 Number Analyzed 1 participants
54.0
Participant-9 Baseline Number Analyzed 1 participants
40.0
Participant-9 Week 312 Number Analyzed 1 participants
52.0
Participant-10 Baseline Number Analyzed 1 participants
48.5
Participant-10 Week 52 Number Analyzed 1 participants
49.5
Participant-11 Baseline Number Analyzed 1 participants
48.0
Participant-11 Week 156 Number Analyzed 1 participants
50.8
Participant-12 Baseline Number Analyzed 1 participants
46.0
Participant-12 Week 468 Number Analyzed 1 participants
56.0
4.Primary Outcome
Title Motor Subscale of Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III) Normative Composite Scores
Hide Description Bayley-III: Instrument designed to measure developmental functioning of infants and toddlers between ages of 1 and 42 months (age adjustments for prematurity are accommodated with tool). Bayley-III administered up to 42 months of age and provides age specific norm-referenced composite scores for cognitive scales (91 items, composite score minimum 55 and maximum 145), language scale (98 items, composite score minimum 47 and maximum 153), motor scale (138 items, composite score minimum 46 and maximum 154) skills. For all raw scores (for scales), higher scores indicates greater number of developmental skills credited. For norm-based composite scales for motor scale, score of 100 defines average performance of given age group, scores of 85 and 115 are 1 standard deviation (SD) below an above mean, respectively, and scores of 70 and 130 are equivalent to 2 SD from mean.
Time Frame Participants 1-12: Baseline, Participant-1: Week 52, Participant-2: Week 83, Participants 3 and 4: Week 104, Participant-6: Week 78, Participants 7 and 8: Week 26, Participant-9: Week 156, Participants 10 and 11: Week 26, Participant-12: Week 104
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: score on a scale
Participant-1 Baseline Number Analyzed 1 participants
94
Participant-1 Week 52 Number Analyzed 1 participants
94
Participant-2 Baseline Number Analyzed 1 participants
94
Participant-2 Week 83 Number Analyzed 1 participants
91
Participant-3 Baseline Number Analyzed 1 participants
46
Participant-3 Week 104 Number Analyzed 1 participants
46
Participant-4 Baseline Number Analyzed 1 participants
46
Participant-4 Week 104 Number Analyzed 1 participants
46
Participant-5 Baseline Number Analyzed 1 participants
58
Participant-6 Baseline Number Analyzed 1 participants
79
Participant-6 Week 78 Number Analyzed 1 participants
100
Participant-7 Baseline Number Analyzed 1 participants
61
Participant-7 Week 26 Number Analyzed 1 participants
46
Participant-8 Baseline Number Analyzed 1 participants
46
Participant-8 Week 26 Number Analyzed 1 participants
46
Participant-9 Baseline Number Analyzed 1 participants
46
Participant-9 Week 156 Number Analyzed 1 participants
70
Participant-10 Baseline Number Analyzed 1 participants
61
Participant-10 Week 26 Number Analyzed 1 participants
73
Participant-11 Baseline Number Analyzed 1 participants
61
Participant-11 Week 26 Number Analyzed 1 participants
58
Participant-12 Baseline Number Analyzed 1 participants
55
Participant-12 Week 104 Number Analyzed 1 participants
61
5.Primary Outcome
Title Gross Motor Function Measure (GMFM-88) Total Scores
Hide Description GMFM-88 is developed specifically to detect quantitative changes in gross motor function that consists of 88 items organized into 5 dimensions: lying and rolling, sitting, crawling and kneeling, standing, and walking, running and jumping. Each item is scored on a 4-point Likert scale that ranges from 0 to 3, i.e., 0=cannot do; 1=initiated (less than [<] 10 percent [%] of task); 2=partially completed (10 to <100% of task); 3=task completion. The score for each dimension is expressed as percentage of the maximum score for that dimension. Total GMFM-88 score is obtained by adding percentage score for each dimension and dividing the sum by total number of dimensions. Total score ranges from 0 to 100, where higher score indicates better gross motor functions.
Time Frame Participants 1-12: Baseline, Participants 1 and 2: Wk 52, Participants 3 and 4: Wk 208, Participant-6: Wk 359, Participant-7: Wk 26, Participant-8: Wk 156, Participant-9: Wk 312, Participant-11: Wk 156, Participant-12: Wk416
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: score on a scale
Participant-1 Baseline Number Analyzed 1 participants
27.98
Participant-1 Week 52 Number Analyzed 1 participants
59.65
Participant-2 Baseline Number Analyzed 1 participants
32.42
Participant-2 Week 52 Number Analyzed 1 participants
91.52
Participant-3 Baseline Number Analyzed 1 participants
7.16
Participant-3 Week 208 Number Analyzed 1 participants
2.75
Participant-4 Baseline Number Analyzed 1 participants
2.18
Participant-4 Week 208 Number Analyzed 1 participants
0.39
Participant-5 Baseline Number Analyzed 1 participants
12.39
Participant-6 Baseline Number Analyzed 1 participants
62.22
Participant-6 Week 359 Number Analyzed 1 participants
97.11
Participant-7 Baseline Number Analyzed 1 participants
8.27
Participant-7 Week 26 Number Analyzed 1 participants
15.83
Participant-8 Baseline Number Analyzed 1 participants
0.39
Participant-8 Week 156 Number Analyzed 1 participants
0.39
Participant-9 Baseline Number Analyzed 1 participants
2.35
Participant-9 Week 312 Number Analyzed 1 participants
88.23
Participant-10 Baseline Number Analyzed 1 participants
34.43
Participant-11 Baseline Number Analyzed 1 participants
16.71
Participant-11 Week 156 Number Analyzed 1 participants
9.86
Participant-12 Baseline Number Analyzed 1 participants
21.94
Participant-12 Week 416 Number Analyzed 1 participants
27.39
6.Primary Outcome
Title Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Scaled Scores
Hide Description Pompe PEDI is disease specific version of PEDI developed to assess functional capabilities and performance in children with Pompe disease from 2 months up to adolescence. It consists of all items of original PEDI Functional Skills Scales and Caregiver Assistance Scales for three content domains: self-care, mobility, and social function. Additional items were added to Functional Skills Scales Mobility and Self-care domains. Norm-based scoring is developed for additional items and scoring algorithms for PEDI are adjusted to reflect normative data collected for Pompe PEDI. Scaled scores for each domain range from 0-100 and provide indication of performance of child along continuum of relatively easy to relatively difficult items in particular domain of PEDI, where higher score indicates increased degrees of functional performance.
Time Frame Participants1-12:Baseline, Participants 1 and 2: Wk 52, Participants 3 and 4: Wk 208, Participant-6: Wk 359, Participant-7: Wk 52, Participant-8: Wk156, Participant-9: Wk 312, Participant-10: Wk 26, Participant-11: Wk 156, Participant-12: Wk 416
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was preformed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoint.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: score on a scale
Participant-1 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
40.62
Participant-1 Week 52: Functional Skills- Self-Care Number Analyzed 1 participants
50.15
Participant-1 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-1 Week 52: Caregiver Assistance- Self-Care Number Analyzed 1 participants
20.1
Participant-1 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
39.68
Participant-1 Week 52: Functional Skills- Mobility Number Analyzed 1 participants
56.31
Participant-1 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
42.7
Participant-1 Week 52: Caregiver Assistance- Mobility Number Analyzed 1 participants
47.2
Participant-1 Baseline: Functional Skills- Social Number Analyzed 1 participants
36.1
Participant-1 Week 52: Functional Skills- Social Number Analyzed 1 participants
45
Participant-1 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
35.9
Participant-1 Week 52: Caregiver Assistance- Social Number Analyzed 1 participants
31.6
Participant-2 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
23.05
Participant-2 Week 52: Functional Skills- Self-Care Number Analyzed 1 participants
59.79
Participant-2 Week 52: Caregiver Assistance- Self-Care Number Analyzed 1 participants
32.3
Participant-2 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
28.81
Participant-2 Week 52: Functional Skills- Mobility Number Analyzed 1 participants
62.11
Participant-2 Week 52: Caregiver Assistance- Mobility Number Analyzed 1 participants
72.7
Participant-2 Week 52: Functional Skills- Social Number Analyzed 1 participants
52
Participant-3 Baseline: Functional Skills-Self-Care Number Analyzed 1 participants
21.03
Participant-3 Week 208: Functional Skills- Self-Care Number Analyzed 1 participants
34.24
Participant-3 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
11.6
Participant-3 Week 208: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-3 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
15.06
Participant-3 Week 208: Functional Skills- Mobility Number Analyzed 1 participants
4.53
Participant-3 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-3 Week 208: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-3 Baseline: Functional Skills- Social Number Analyzed 1 participants
21.6
Participant-3 Week 208: Functional Skills- Social Number Analyzed 1 participants
40.4
Participant-3 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-3 Week 208: Caregiver Assistance- Social Number Analyzed 1 participants
11.3
Participant-4 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
4.92
Participant-4 Week 208: Functional Skills- Self-Care Number Analyzed 1 participants
23.05
Participant-4 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-4 Week 208: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-4 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
4.53
Participant-4 Week 208: Functional Skills- Mobility Number Analyzed 1 participants
0
Participant-4 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-4 Week 208: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-4 Baseline: Functional Skills- Social Number Analyzed 1 participants
14.7
Participant-4 Week 208: Functional Skills-Social Number Analyzed 1 participants
40.4
Participant-4 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-4 Week 208: Caregiver Assistance- Social Number Analyzed 1 participants
11.3
Participant-5 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
36.16
Participant-5 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-5 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
19.75
Participant-5 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-5 Baseline: Functional Skills- Social Number Analyzed 1 participants
32.9
Participant-5 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-6 Baseline: Functional Skills-Self-Care Number Analyzed 1 participants
45.7
Participant-6 Week 359: Functional Skills-Self-Care Number Analyzed 1 participants
81.36
Participant-6 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
44.4
Participant-6 Week 359: Caregiver Assistance- Self-Care Number Analyzed 1 participants
100
Participant-6 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
52.44
Participant-6 Week 359: Functional Skills- Mobility Number Analyzed 1 participants
76.46
Participant-6 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
61.8
Participant-6 Week 359: Caregiver Assistance- Mobility Number Analyzed 1 participants
100
Participant-6 Baseline: Functional Skills- Social Number Analyzed 1 participants
40.4
Participant-6 Week 359: Functional Skills-Social Number Analyzed 1 participants
100
Participant-6 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
57.3
Participant-6 Week 359: Caregiver Assistance- Social Number Analyzed 1 participants
100
Participant-7 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
12.7
Participant-7 Week 52: Functional Skills-Self-Care Number Analyzed 1 participants
33.1
Participant-7 Week 52: Caregiver Assistance- Self-Care Number Analyzed 1 participants
11.6
Participant-7 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
4.53
Participant-7 Week 52: Functional Skills- Mobility Number Analyzed 1 participants
25.12
Participant-7 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-7 Week 52: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-7 Baseline: Functional Skills- Social Number Analyzed 1 participants
6.6
Participant-7 Week 52: Functional Skills-Social Number Analyzed 1 participants
27.7
Participant-7 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-7 Week 52: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-8 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
0.01
Participant-8 Week 156: Functional Skills- Self-Care Number Analyzed 1 participants
12.7
Participant-8 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-8 Week 156: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-8 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
0.01
Participant-8 Week 156: Functional Skills- Mobility Number Analyzed 1 participants
0
Participant-8 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-8 Week 156: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-8 Baseline: Functional Skills- Social Number Analyzed 1 participants
3.1
Participant-8 Week 156: Functional Skills-Social Number Analyzed 1 participants
35.1
Participant-8 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-8 Week 156: Caregiver Assistance- Social Number Analyzed 1 participants
20.4
Participant-9 Baseline: Functional Skills-Self-Care Number Analyzed 1 participants
16.09
Participant-9 Week 312: Functional Skills-Self-Care Number Analyzed 1 participants
77.1
Participant-9 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
0
Participant-9 Week 312: Caregiver Assistance- Self-Care Number Analyzed 1 participants
100
Participant-9 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
4.53
Participant-9 Week 312: Functional Skills- Mobility Number Analyzed 1 participants
63.25
Participant-9 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-9 Week 312 :Caregiver Assistance- Mobility Number Analyzed 1 participants
100
Participant-9 Baseline: Functional Skills- Social Number Analyzed 1 participants
10.5
Participant-9 Week 312: Functional Skills-Social Number Analyzed 1 participants
73.4
Participant-9 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
0
Participant-9 Week 312: Caregiver Assistance- Social Number Analyzed 1 participants
100
Participant-10 Baseline: Functional Skills-Self-Care Number Analyzed 1 participants
35.25
Participant-10 Week 26: Functional Skills- Self-Care Number Analyzed 1 participants
49.18
Participant-10 Baseline: Caregiver Assistance Self-Care Number Analyzed 1 participants
11.6
Participant-10 Week 26: Caregiver Assistance- Self-Care Number Analyzed 1 participants
42.8
Participant-10 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
34.55
Participant-10 Week 26: Functional Skills- Mobility Number Analyzed 1 participants
49.26
Participant-10 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
31.9
Participant-10 Week 26: Caregiver Assistance- Mobility Number Analyzed 1 participants
48.5
Participant-10 Baseline: Functional Skills- Social Number Analyzed 1 participants
31.6
Participant-10 Week 26: Functional Skills- Social Number Analyzed 1 participants
44.4
Participant-10 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
11.3
Participant-10 Week 26: Caregiver Assistance- Social Number Analyzed 1 participants
50.9
Participant-11 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
43.58
Participant-11 Week 156: Functional Skills- Self-Care Number Analyzed 1 participants
43
Participant-11 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
25.4
Participant-11 Week 156: Caregiver Assistance- Self-Care Number Analyzed 1 participants
25.4
Participant-11 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
28.15
Participant-11 Week 156: Functional Skills- Mobility Number Analyzed 1 participants
25.93
Participant-11 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
0
Participant-11 Week 156: Caregiver Assistance- Mobility Number Analyzed 1 participants
11.7
Participant-11 Baseline: Functional Skills-Social Number Analyzed 1 participants
37
Participant-11 Week 156: Functional Skills- Social Number Analyzed 1 participants
51.4
Participant-11 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
11.3
Participant-11 Week 156: Caregiver Assistance- Social Number Analyzed 1 participants
39.6
Participant-12 Baseline: Functional Skills- Self-Care Number Analyzed 1 participants
31.8
Participant-12 Week 416: Functional Skills-Self-Care Number Analyzed 1 participants
64.08
Participant-12 Baseline: Caregiver Assistance- Self-Care Number Analyzed 1 participants
11.6
Participant-12 Week 416: Caregiver Assistance- Self-Care Number Analyzed 1 participants
55.7
Participant-12 Baseline: Functional Skills- Mobility Number Analyzed 1 participants
26.71
Participant-12 Week 416: Functional Skills- Mobility Number Analyzed 1 participants
48.06
Participant-12 Baseline: Caregiver Assistance- Mobility Number Analyzed 1 participants
11.7
Participant-12 Week 416: Caregiver Assistance- Mobility Number Analyzed 1 participants
40.9
Participant-12 Baseline: Functional Skills- Social Number Analyzed 1 participants
37.9
Participant-12 Week 416: Functional Skills- Social Number Analyzed 1 participants
73.4
Participant-12 Baseline: Caregiver Assistance- Social Number Analyzed 1 participants
11.3
Participant-12 Week 416: Caregiver Assistance- Social Number Analyzed 1 participants
63.3
7.Primary Outcome
Title Cognitive and Language Subscales of Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III) Normative Composite Scores
Hide Description Bayley-III: Instrument designed to measure developmental functioning of infants and toddlers between ages of 1 and 42 months (age adjustments for prematurity are accommodated with tool). Bayley-III administered up to 42 months of age and provides age specific norm-referenced composite scores for cognitive scales (91 items, composite score minimum 55 and maximum 145), language scale (98 items, composite score minimum 47 and maximum 153), motor scale (138 items, composite score minimum 46 and maximum 154) skills. For all raw scores (for scales), higher scores indicates greater number of developmental skills credited. For norm-based composite scales for cognitive and language, score of 100 defines average performance of given age group, scores of 85 and 115 are 1 SD below an above mean, respectively, and scores of 70 and 130 are equivalent to 2 SD from mean.
Time Frame Participants 1-12: Baseline, Participant-1: Week 52, Participant-2: Week 83, Participants 3 and 4: Week 104, Participant-6: Week 78, Participants 7 and 8: Week 26, Participant-9: Week 156, Participants 10 and 11:Week 26, Participant-12: Week 104
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Number
Unit of Measure: score on a scale
Participant-1 Baseline: Cognitive Number Analyzed 1 participants
90
Participant-1 Week 52: Cognitive Number Analyzed 1 participants
100
Participant-2 Baseline: Cognitive Number Analyzed 1 participants
85
Participant-2 Week 83: Cognitive Number Analyzed 1 participants
100
Participant-3 Baseline: Cognitive Number Analyzed 1 participants
65
Participant-3 Week 104: Cognitive Number Analyzed 1 participants
55
Participant-4 Baseline: Cognitive Number Analyzed 1 participants
55
Participant-4 Week 104: Cognitive Number Analyzed 1 participants
55
Participant-5 Baseline: Cognitive Number Analyzed 1 participants
60
Participant-6 Baseline: Cognitive Number Analyzed 1 participants
75
Participant-6 Week 78: Cognitive Number Analyzed 1 participants
105
Participant-7 Baseline: Cognitive Number Analyzed 1 participants
65
Participant-7 Week 26: Cognitive Number Analyzed 1 participants
60
Participant-8 Baseline: Cognitive Number Analyzed 1 participants
55
Participant-8 Week 26: Cognitive Number Analyzed 1 participants
55
Participant-9 Baseline: Cognitive Number Analyzed 1 participants
55
Participant-9 Week 156: Cognitive Number Analyzed 1 participants
65
Participant-10 Baseline: Cognitive Number Analyzed 1 participants
85
Participant-10 Week 26: Cognitive Number Analyzed 1 participants
85
Participant-11 Baseline: Cognitive Number Analyzed 1 participants
75
Participant-11 Week 26: Cognitive Number Analyzed 1 participants
75
Participant-12 Baseline: Cognitive Number Analyzed 1 participants
80
Participant-12 Week 104: Cognitive Number Analyzed 1 participants
85
Participant-1 Baseline: Language Number Analyzed 1 participants
103
Participant-1 Week 52: Language Number Analyzed 1 participants
103
Participant-2 Baseline: Language Number Analyzed 1 participants
79
Participant-2 Week 83: Language Number Analyzed 1 participants
103
Participant-3 Baseline: Language Number Analyzed 1 participants
59
Participant-3 Week 104: Language Number Analyzed 1 participants
53
Participant-4 Baseline: Language Number Analyzed 1 participants
47
Participant-4 Week 104: Language Number Analyzed 1 participants
47
Participant-5 Baseline: Language Number Analyzed 1 participants
71
Participant-6 Baseline: Language Number Analyzed 1 participants
71
Participant-6 Week 78: Language Number Analyzed 1 participants
91
Participant-7 Baseline: Language Number Analyzed 1 participants
65
Participant-7 Week 26: Language Number Analyzed 1 participants
50
Participant-8 Baseline: Language Number Analyzed 1 participants
47
Participant-8 Week 26: Language Number Analyzed 1 participants
47
Participant-9 Baseline: Language Number Analyzed 1 participants
53
Participant-9 Week 156: Language Number Analyzed 1 participants
74
Participant-10 Baseline: Language Number Analyzed 1 participants
94
Participant-10 Week 26: Language Number Analyzed 1 participants
86
Participant-11 Baseline: Language Number Analyzed 1 participants
68
Participant-11 Week 26: Language Number Analyzed 1 participants
71
Participant-12 Baseline: Language Number Analyzed 1 participants
86
Participant-12 Week 104: Language Number Analyzed 1 participants
65
8.Primary Outcome
Title Brief Intelligence Quotient (IQ) Score of the Leiter International Performance Scale-Revised (Leiter-R)
Hide Description Leiter Scale is designed as nonverbal measure of intellectual function, memory and attention for Participants with communication disorders, hearing impairments, motor impairments, certain types of learning disabilities. Leiter-R was administered to participants after aging out of Bayley-III (at 42 months of age) and before Leiter-3 utilization (per protocol, due to discontinuation of Leiter-R). Leiter-R scale consists of 2 groups of subtests: Visualization-Reasoning Battery, Attention-Memory Battery. Subtests in Leiter-R were Figure Ground, Form Completion, Sequential Order, Repeated Patterns using that 'Brief Scale IQ' was scored for estimation of intellectual ability. Brief-IQ scores range is 30-170, where higher scores indicates higher intelligence. Score of 100 is expected mean standard score at each age interval. 95% children in each age group (based on normative sample) are expected to score within 2 SD of mean.
Time Frame Participants 1 and 2: Week 156, Participant-3: Week 260, Participant-4: Week 156, Participant-5: Week 208
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints (most recent visit). Here, "Overall number of participants analyzed" signifies participants who were evaluated for this outcome measure.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: score on a scale
Participant-1 Week 156 Number Analyzed 1 participants
54
Participant-2 Week 156 Number Analyzed 1 participants
50
Participant-3 Week 260 Number Analyzed 1 participants
100
Participant-4 Week 156 Number Analyzed 1 participants
98
Participant-5 Week 208 Number Analyzed 1 participants
97
9.Primary Outcome
Title Nonverbal Intelligence Quotient (IQ) Score of Leiter International Performance Scale - 3rd Edition (Leiter-3)
Hide Description Leiter Scale: Designed as nonverbal measure of intellectual function, memory and attention for participants with communication disorders, hearing impairments, motor impairments, certain types of learning disabilities. Leiter-3 has 2 groups of subtests: cognitive battery and attention/memory battery. Nonverbal intelligence estimates global intellectual ability. The 4 cognitive battery subtests are: Figure Ground, Form Completion, Sequential Order, Classification-analogies along with 1 optional subset, Visual Patterns. Nonverbal IQ scores range is 30-170, which encompass 'severe delay' to 'extremely high/gifted', higher numbers indicates higher intelligence. Score of 100 is expected mean standard score at each age interval. 95% children in each age group (based on normative sample) are expected to score within 2 SD of mean.
Time Frame Participant-1: Week 156, Participant-2: Week 312, Participant-3: Week 416
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS population. No summary analysis was done and participant wise data were reported at available specified timepoints (most recent visit). Here, "Overall number of participants analyzed" signifies number of participants who were evaluated for this outcome measure.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: score on a scale
Participant-1 Week 156 Number Analyzed 1 participants
87
Participant-2 Week 312 Number Analyzed 1 participants
78
Participant-3 Week 416 Number Analyzed 1 participants
70
10.Secondary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Hide Description Adverse event (AE): any undesirable physical, psychological or behavioral effect experienced by participants during their participation in an investigational study, in conjunction with the use of the drug or biologic, whether or not product-related. Any untoward signs or symptoms experienced by the participant from the time of signing of the informed consent until completion of the study. Serious AE (SAE): any AE that resulted in any of the following outcomes: death, life-threatening experience, required hospitalization or prolonged inpatient hospitalization, persistent or significant disability/incapacity, congenital anomaly, and important medical events. TEAEs: AEs that developed, worsened, or became serious during the treatment-emergent period (defined as the period from the first study drug administration until last study assessment).
Time Frame From Baseline up to 13.25 years
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks and were followed for 10 years or up to discontinuation from study treatment due to any reason.
Overall Number of Participants Analyzed 12
Measure Type: Count of Participants
Unit of Measure: Participants
TEAEs
11
  91.7%
TESAEs
9
  75.0%
Time Frame From administration of first dose of study drug up to 13.25 years
Adverse Event Reporting Description Reported AEs and deaths are TEAEs that developed, worsened, or became serious during the treatment period (from the first administration of study drug in the study to the last study assessment). Analysis was performed on FAS population.
 
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Participants received alglucosidase alfa 20 mg/kg body weight as intravenous infusion every 2 weeks until 10 years of age or up to discontinuation from study treatment due to any reason.
All-Cause Mortality
Alglucosidase Alfa
Affected / at Risk (%)
Total   3/12 (25.00%)    
Hide Serious Adverse Events
Alglucosidase Alfa
Affected / at Risk (%) # Events
Total   9/12 (75.00%)    
Cardiac disorders   
Cardio-Respiratory Arrest † 1  1/12 (8.33%)  1
Tachycardia † 1  1/12 (8.33%)  1
Congenital, familial and genetic disorders   
Glycogen Storage Disease Type Ii † 1  1/12 (8.33%)  1
Gastrointestinal disorders   
Dysphagia † 1  1/12 (8.33%)  1
General disorders   
Pyrexia † 1  2/12 (16.67%)  2
Infections and infestations   
Bacteraemia † 1  1/12 (8.33%)  2
Bronchitis † 1  1/12 (8.33%)  1
Catheter Site Infection † 1  1/12 (8.33%)  1
Device Related Sepsis † 1  1/12 (8.33%)  2
Gastroenteritis † 1  1/12 (8.33%)  1
Medical Device Site Infection † 1  1/12 (8.33%)  1
Pneumonia † 1  7/12 (58.33%)  18
Pneumonia Aspiration † 1  3/12 (25.00%)  4
Pneumonia Bacterial † 1  1/12 (8.33%)  1
Pseudomonas Infection † 1  1/12 (8.33%)  1
Respiratory Syncytial Virus Bronchiolitis † 1  1/12 (8.33%)  1
Sepsis † 1  1/12 (8.33%)  1
Vascular Device Infection † 1  5/12 (41.67%)  5
Investigations   
Oxygen Saturation Decreased † 1  1/12 (8.33%)  1
Metabolism and nutrition disorders   
Acidosis † 1  1/12 (8.33%)  1
Diabetes Mellitus † 1  1/12 (8.33%)  1
Feeding Disorder † 1  1/12 (8.33%)  1
Nervous system disorders   
Hypoglycaemic Seizure † 1  2/12 (16.67%)  2
Neurological Decompensation † 1  1/12 (8.33%)  1
Seizure † 1  1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders   
Apnoea † 1  1/12 (8.33%)  1
Aspiration † 1  1/12 (8.33%)  1
Atelectasis † 1  4/12 (33.33%)  4
Bronchial Secretion Retention † 1  1/12 (8.33%)  1
Chronic Respiratory Failure † 1  1/12 (8.33%)  1
Dyspnoea † 1  1/12 (8.33%)  1
Hypercapnia † 1  1/12 (8.33%)  1
Hypoxia † 1  2/12 (16.67%)  4
Pneumonitis † 1  1/12 (8.33%)  1
Pulmonary Hypertension † 1  1/12 (8.33%)  1
Respiratory Distress † 1  4/12 (33.33%)  4
Respiratory Failure † 1  5/12 (41.67%)  8
Velopharyngeal Incompetence † 1  1/12 (8.33%)  1
Wheezing † 1  1/12 (8.33%)  1
Skin and subcutaneous tissue disorders   
Dermatitis Contact † 1  1/12 (8.33%)  1
Vascular disorders   
Hypotension † 1  1/12 (8.33%)  1
1
Term from vocabulary, MedDRA 24.1
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Alglucosidase Alfa
Affected / at Risk (%) # Events
Total   11/12 (91.67%)    
Blood and lymphatic system disorders   
Iron Deficiency Anaemia † 1  1/12 (8.33%)  1
Cardiac disorders   
Right Ventricular Hypertrophy † 1  1/12 (8.33%)  1
Sinus Tachycardia † 1  1/12 (8.33%)  1
Tachycardia † 1  3/12 (25.00%)  17
Ear and labyrinth disorders   
Deafness † 1  1/12 (8.33%)  1
Deafness Neurosensory † 1  4/12 (33.33%)  4
External Ear Disorder † 1  1/12 (8.33%)  1
Middle Ear Effusion † 1  1/12 (8.33%)  1
Eye disorders   
Chalazion † 1  1/12 (8.33%)  1
Eye Discharge † 1  2/12 (16.67%)  2
Eye Swelling † 1  1/12 (8.33%)  1
Eyelid Ptosis † 1  2/12 (16.67%)  2
Papilloedema † 1  2/12 (16.67%)  2
Visual Acuity Reduced † 1  1/12 (8.33%)  1
Gastrointestinal disorders   
Constipation † 1  2/12 (16.67%)  2
Dental Caries † 1  1/12 (8.33%)  1
Diarrhoea † 1  4/12 (33.33%)  6
Gingival Swelling † 1  1/12 (8.33%)  1
Mouth Ulceration † 1  1/12 (8.33%)  1
Post-Tussive Vomiting † 1  1/12 (8.33%)  1
Salivary Hypersecretion † 1  1/12 (8.33%)  1
Swollen Tongue † 1  1/12 (8.33%)  1
Teething † 1  1/12 (8.33%)  2
Vomiting † 1  7/12 (58.33%)  11
General disorders   
Asthenia † 1  2/12 (16.67%)  2
Catheter Site Rash † 1  1/12 (8.33%)  1
Mass † 1  1/12 (8.33%)  1
Pain † 1  4/12 (33.33%)  6
Pyrexia † 1  9/12 (75.00%)  42
Swelling † 1  1/12 (8.33%)  1
Swelling Face † 1  1/12 (8.33%)  1
Unevaluable Event † 1  1/12 (8.33%)  1
Vascular Device Occlusion † 1  1/12 (8.33%)  1
Immune system disorders   
Allergy To Animal † 1  1/12 (8.33%)  1
Multiple Allergies † 1  1/12 (8.33%)  1
Infections and infestations   
Ear Infection † 1  2/12 (16.67%)  2
Fungal Skin Infection † 1  1/12 (8.33%)  1
Influenza † 1  2/12 (16.67%)  2
Oral Candidiasis † 1  1/12 (8.33%)  2
Otitis Media † 1  6/12 (50.00%)  11
Pharyngitis Streptococcal † 1  1/12 (8.33%)  2
Pneumonia † 1  6/12 (50.00%)  9
Rash Pustular † 1  1/12 (8.33%)  1
Respiratory Tract Infection † 1  1/12 (8.33%)  1
Rhinitis † 1  1/12 (8.33%)  2
Upper Respiratory Tract Infection † 1  5/12 (41.67%)  13
Urinary Tract Infection † 1  2/12 (16.67%)  4
Viral Pharyngitis † 1  1/12 (8.33%)  1
Viral Upper Respiratory Tract Infection † 1  1/12 (8.33%)  2
Vulvovaginal Candidiasis † 1  1/12 (8.33%)  1
Injury, poisoning and procedural complications   
Anaesthetic Complication † 1  1/12 (8.33%)  1
Femur Fracture † 1  1/12 (8.33%)  1
Joint Dislocation † 1  1/12 (8.33%)  1
Procedural Pain † 1  1/12 (8.33%)  1
Stoma Site Haemorrhage † 1  1/12 (8.33%)  1
Investigations   
Audiogram Abnormal † 1  1/12 (8.33%)  1
Bacterial Test Positive † 1  1/12 (8.33%)  1
Blood Potassium Decreased † 1  1/12 (8.33%)  1
Blood Urine Present † 1  1/12 (8.33%)  1
Body Temperature Increased † 1  2/12 (16.67%)  2
Clostridium Test Positive † 1  1/12 (8.33%)  2
Electrocardiogram Qrs Complex Prolonged † 1  1/12 (8.33%)  1
Electrocardiogram Qt Prolonged † 1  1/12 (8.33%)  1
Electrocardiogram T Wave Inversion † 1  1/12 (8.33%)  1
Fungal Test Positive † 1  1/12 (8.33%)  1
Haematocrit Decreased † 1  1/12 (8.33%)  1
Haemoglobin Decreased † 1  1/12 (8.33%)  1
Oxygen Saturation Decreased † 1  1/12 (8.33%)  5
Pseudomonas Test Positive † 1  1/12 (8.33%)  1
Tympanometry Abnormal † 1  1/12 (8.33%)  1
Urine Output Decreased † 1  1/12 (8.33%)  1
Metabolism and nutrition disorders   
Dehydration † 1  2/12 (16.67%)  2
Diabetes Mellitus † 1  1/12 (8.33%)  1
Feeding Disorder † 1  2/12 (16.67%)  2
Hypoglycaemia † 1  1/12 (8.33%)  1
Hypokalaemia † 1  1/12 (8.33%)  5
Hyponatraemia † 1  1/12 (8.33%)  1
Hypophosphataemia † 1  1/12 (8.33%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia † 1  3/12 (25.00%)  3
Extremity Contracture † 1  2/12 (16.67%)  2
Foot Deformity † 1  1/12 (8.33%)  1
Joint Contracture † 1  2/12 (16.67%)  2
Muscular Weakness † 1  2/12 (16.67%)  3
Myopathy † 1  1/12 (8.33%)  1
Pain In Extremity † 1  1/12 (8.33%)  1
Scoliosis † 1  1/12 (8.33%)  1
Spinal Deformity † 1  1/12 (8.33%)  1
Nervous system disorders   
Areflexia † 1  1/12 (8.33%)  1
Encephalopathy † 1  1/12 (8.33%)  1
Focal Dyscognitive Seizures † 1  1/12 (8.33%)  1
Hypotonia † 1  2/12 (16.67%)  2
Seizure † 1  1/12 (8.33%)  1
White Matter Lesion † 1  1/12 (8.33%)  1
Product Issues   
Device Malfunction † 1  3/12 (25.00%)  4
Renal and urinary disorders   
Nephrolithiasis † 1  1/12 (8.33%)  1
Reproductive system and breast disorders   
Balanoposthitis † 1  1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders   
Aspiration † 1  1/12 (8.33%)  1
Asthma † 1  2/12 (16.67%)  2
Atelectasis † 1  5/12 (41.67%)  7
Bronchial Secretion Retention † 1  1/12 (8.33%)  1
Bronchospasm † 1  1/12 (8.33%)  1
Cough † 1  8/12 (66.67%)  28
Dysphonia † 1  1/12 (8.33%)  1
Dyspnoea † 1  3/12 (25.00%)  5
Epistaxis † 1  1/12 (8.33%)  1
Hypercapnia † 1  2/12 (16.67%)  3
Hypoventilation † 1  1/12 (8.33%)  2
Hypoxia † 1  1/12 (8.33%)  1
Increased Upper Airway Secretion † 1  1/12 (8.33%)  1
Nasal Congestion † 1  2/12 (16.67%)  2
Oropharyngeal Pain † 1  1/12 (8.33%)  1
Pleural Effusion † 1  1/12 (8.33%)  1
Productive Cough † 1  1/12 (8.33%)  1
Pulmonary Oedema † 1  1/12 (8.33%)  1
Respiratory Distress † 1  1/12 (8.33%)  1
Respiratory Failure † 1  3/12 (25.00%)  3
Respiratory Tract Congestion † 1  1/12 (8.33%)  1
Rhinorrhoea † 1  5/12 (41.67%)  16
Rhonchi † 1  1/12 (8.33%)  2
Sinus Congestion † 1  2/12 (16.67%)  2
Sneezing † 1  1/12 (8.33%)  2
Stridor † 1  1/12 (8.33%)  1
Upper Respiratory Tract Congestion † 1  1/12 (8.33%)  3
Velopharyngeal Incompetence † 1  1/12 (8.33%)  1
Wheezing † 1  3/12 (25.00%)  4
Skin and subcutaneous tissue disorders   
Angioedema † 1  1/12 (8.33%)  2
Decubitus Ulcer † 1  1/12 (8.33%)  1
Dermatitis Diaper † 1  3/12 (25.00%)  5
Dry Skin † 1  2/12 (16.67%)  2
Erythema † 1  3/12 (25.00%)  3
Hair Growth Abnormal † 1  2/12 (16.67%)  2
Papule † 1  1/12 (8.33%)  1
Rash † 1  4/12 (33.33%)  20
Rash Papular † 1  1/12 (8.33%)  1
Skin Disorder † 1  1/12 (8.33%)  1
Urticaria † 1  3/12 (25.00%)  19
Vascular disorders   
Flushing † 1  2/12 (16.67%)  2
Hypotension † 1  1/12 (8.33%)  1
1
Term from vocabulary, MedDRA 24.1
†
Indicates events were collected by systematic assessment
No summary analysis was done and participant wise data were reported at available specified timepoints. Reporting of participant numbers is per-endpoint and not consistent between assessments to maintain participant's privacy.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Trial Transparency Team
Organization: Sanofi
Phone: 800-633-1610 ext 6#
EMail: Contact-US@sanofi.com
Layout table for additonal information
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00486889    
Other Study ID Numbers: AGLU03606
LTS12869 ( Other Identifier: Sanofi )
U1111-1163-0368 ( Other Identifier: UTN )
2021-005552-11 ( EudraCT Number )
First Submitted: June 13, 2007
First Posted: June 15, 2007
Results First Submitted: July 29, 2022
Results First Posted: August 26, 2022
Last Update Posted: August 26, 2022