Trial Outcomes & Findings for An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease (NCT NCT00701129)

NCT ID: NCT00701129

Last Updated: 2014-05-13

Results Overview

Serum samples from patients were analyzed for the presence of anti-rhGAA IgG antibodies. End of study (EOS) refers to the last post baseline observation during study period (up to Week 79).

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 4 participants
• Primary outcome timeframe: Baseline, End of Study (up to Week 79 or early termination)
• Results posted on: 2014-05-13

Participant Flow

The study was conducted at 2 centers in the United States of America between October 01, 2009 and March 27, 2013.

Participant milestones

Measure	Alglucosidase Alfa Alglucosidase alfa (Myozyme®) 20 milligrams per kilogram (mg/kg) intravenous (IV) infusion every other week (qow) (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was less than (\<) 6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 milligrams per square meter (mg/m\^2) (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Overall Study STARTED	4
Overall Study Full Analysis Set (FAS)	4
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure

Alglucosidase Alfa Alglucosidase alfa (Myozyme®) 20 milligrams per kilogram (mg/kg) intravenous (IV) infusion every other week (qow) (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was less than (\<) 6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 milligrams per square meter (mg/m\^2) (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.

Overall Study Death

2

Baseline Characteristics

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Baseline characteristics by cohort

Measure	Alglucosidase Alfa n=4 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Age, Customized Less Than or Equal to (=<) 6 Months	2 participants n=5 Participants
Age, Customized Greater Than (>) 6 Months	2 participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Race/Ethnicity, Customized Race: Black	2 participants n=5 Participants
Race/Ethnicity, Customized Race: White	1 participants n=5 Participants
Race/Ethnicity, Customized Race: White, Black	1 participants n=5 Participants
Race/Ethnicity, Customized Ethnicity: Hispanic	2 participants n=5 Participants
Race/Ethnicity, Customized Ethnicity: Non Hispanic	2 participants n=5 Participants
Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G Negative	4 participants n=5 Participants
Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G Positive	0 participants n=5 Participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index LVM Z-score: Normal	0 participants n=5 Participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index LVM Z-score: Abnormal	4 participants n=5 Participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index LVM index: Normal	0 participants n=5 Participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index LVM index: Abnormal	4 participants n=5 Participants
Number of Patients With Ventilator Use Yes	3 participants n=5 Participants
Number of Patients With Ventilator Use No	1 participants n=5 Participants
Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88)	5.10 percentage of maximum total score n=5 Participants
Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS)	0 months n=5 Participants
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Functional Skills: Mobility Score (n=4)	4.5 units on a scale n=5 Participants
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Functional Skills: Self-Care Score (n=4)	14.4 units on a scale n=5 Participants
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Functional Skills: Social Function Score (n=1)	10.5 units on a scale n=5 Participants
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Caregiver Assistance: Mobility Score (n=2)	0 units on a scale n=5 Participants
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Caregiver Assistance: Self-Care Score (n=2)	0 units on a scale n=5 Participants
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) Caregiver Assistance: Social Function Score (n=2)	0 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: Baseline, End of Study (up to Week 79 or early termination)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa.

Serum samples from patients were analyzed for the presence of anti-rhGAA IgG antibodies. End of study (EOS) refers to the last post baseline observation during study period (up to Week 79).

Outcome measures

Measure	Alglucosidase Alfa n=4 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Change From Baseline in Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study Baseline - Negative; EOS - Positive	2 participants
Change From Baseline in Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study Baseline - Negative; EOS - Negative	2 participants

PRIMARY outcome

Timeframe: End of study (up to Week 79)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, number of patients analyzed = patients with positive anti-rhGAA IgG antibody.

Patients with positive anti-rhGAA IgG antibody were assessed for the presence of inhibitory antibodies (inhibition of enzyme activity and inhibition of enzyme uptake). Enzyme-linked immunosorbent assay (ELISA) was used to measure inhibition of rhGAA enzymatic activity in vitro and a cell-based assay was used to measure the inhibition of the uptake of rhGAA in normal fibroblast cells by flow cytometry.

Outcome measures

Measure	Alglucosidase Alfa n=2 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Number of Patients With Recombinant Human Acid Alfa-glucosidase (rhGAA) Inhibitory Antibody at End of Study Inhibition of Enzyme Activity	0 participants
Number of Patients With Recombinant Human Acid Alfa-glucosidase (rhGAA) Inhibitory Antibody at End of Study Inhibition of Enzyme Uptake	0 participants

PRIMARY outcome

Timeframe: Baseline up to End of study (Week 79)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa.

Outcome measures

Measure

Alglucosidase Alfa n=4 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.

Number of Patients Who Survived at End of Study

2 participants

PRIMARY outcome

Timeframe: End of study (up to Week 79 or early termination)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa.

LVM Z-score and LVM index were assessed by ECHO. LVM Z-Score provides an indicator of degree of standard deviations from the mean in a normal distribution. Negative values indicate a smaller LVM than mean and values higher than 0 indicate a larger LVM than the mean. The normal range for LVM Z-Score is -2 to 2. Values <-2 or >2 indicate abnormal LVM Z-Score. LVM index is an index value derived by normalizing LVM by body surface area. LVM index provides evidence of cardiomyopathy. LVM index values <65 gram per meter^2 (g/m^2) were considered as normal and LVM index values >=65 g/m^2 were considered as abnormal. End of study refers to the last post baseline observation during study period (up to Week 79).

Outcome measures

Measure	Alglucosidase Alfa n=4 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study LVM Z-score: Normal	3 participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study LVM Z-score: Abnormal	1 participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study LVM index: Normal	2 participants
Number of Patients With Normal/Abnormal Left Ventricular Mass (LVM) Z-Score and LVM Index at End of Study LVM index: Abnormal	2 participants

PRIMARY outcome

Timeframe: End of study (up to Week 79 or early termination)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa.

Number of patients who had ventilator support at end of study was reported. End of study refers to the last post baseline observation during study period (up to Week 79).

Outcome measures

Measure	Alglucosidase Alfa n=4 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Number of Patients With Ventilator Use at End of Study No	1 participants
Number of Patients With Ventilator Use at End of Study Yes	3 participants

PRIMARY outcome

Timeframe: End of study (up to Week 79 or early termination)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, numbers of patients analyzed = patients with end of study GMFM-88 assessment.

GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item is scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension is expressed as a percentage of the maximum score for that dimension. Total score is obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. A total score of <7.5% demonstrates gross motor disability. End of study refers to the last post baseline observation during study period (up to Week 79).

Outcome measures

Measure

Alglucosidase Alfa n=3 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.

Gross Motor Disability Assessed by Gross Motor Function Measure-88 (GMFM-88) at End of Study

8.24 percentage of maximum total score Interval 6.76 to 89.8

PRIMARY outcome

Timeframe: End of study (up to Week 79 or early termination)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, numbers of patients analyzed = patients with end of study AIMS assessment.

AIMS is a 58-item reliable and valid measure of motor development for infants at risk for motor delay. It assesses infant movement in 4 positions (subscales): prone (reciprocal crawling); supine (moving hands to feet); sitting (sitting with arm support); and standing (pulls to stand). For each subscale, items were scored as "observed" or "not observed". Item in the observed range create a motor window. When scoring, subscale scores are calculated by giving the child credit (1 point) for observed items within the motor window in addition to being given credit (1 point) for all of the less mature items before motor window. AIMS total score was calculated by summing the scores for 58 items and ranged from 0 to 58, with higher score indicating more mature motor development. Score was then compared with age-equivalent peers from normative sample and equivalence level age (in months) is reported. End of study refers to the last post baseline observation during study period (up to Week 79).

Outcome measures

Measure

Alglucosidase Alfa n=3 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.

Motor Development Status Assessed by Alberta Infantile Motor Scale (AIMS) at End of Study

1.09 months Interval 1.0 to 14.5

PRIMARY outcome

Timeframe: End of study (up to Week 79 or early termination)

Population: FAS population included all enrolled patients who signed informed consent and received at least 1 dose of alglucosidase alfa. Here, number of patient analyzed = number of patients with end of study Pompe PEDI assessment and n = number of patients with end of study assessment of specified category.

The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. It consists of all items of the original PEDI (197 functional skill items in 3 domains: self-care; mobility; and social function) and additional items in the functional skills, mobility, and self-care domains to reflect clinically relevant functional skills for children with Pompe disease. Each domain consisted of 2 subdomains: functional skill performance and caregiver assistance scale. Norm-based scoring was developed for these additional items, and scoring algorithms for the PEDI have been adjusted to reflect additional normative data collected for the Pompe PEDI. Total score range for each domain (mean of subdomains) and subdomain ranges from 0 to 100, where higher score indicates high capability. End of study refers to the last post baseline observation during study period (up to Week 79).

Outcome measures

Measure	Alglucosidase Alfa n=3 Participants Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study Functional Skills: Mobility Score (n=3)	25.1 units on a scale Interval 23.2 to 56.0
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study Functional Skills: Self-Care Score (n=3)	39.3 units on a scale Interval 37.0 to 55.2
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study Functional Skills: Social Function Score (n=3)	46.2 units on a scale Interval 40.4 to 46.8
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study Caregiver Assistance: Mobility Score (n=3)	20.3 units on a scale Interval 20.3 to 31.9
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study Caregiver Assistance: Self-Care Score (n=2)	28.7 units on a scale Interval 20.1 to 37.2
Disability Index Assessed by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at End of Study Caregiver Assistance: Social Function Score (n=3)	48.5 units on a scale Interval 20.4 to 53.1

Adverse Events

Alglucosidase Alfa

Serious events: 3 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Measure	Alglucosidase Alfa n=4 participants at risk Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Cardiac disorders Arrhythmia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Bradycardia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Cardiac arrest	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Cardiopulmonary failure	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Ear and labyrinth disorders Tympanic membrane disorder	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Disease progression	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Pyrexia	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Clostridium difficile colitis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Pneumonia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Serratia sepsis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Tracheitis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Feeding tube complication	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Torus fracture	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Pulse absent	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Metabolism and nutrition disorders Fluid imbalance	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Musculoskeletal and connective tissue disorders Muscle contracture	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Apnoea	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Dyspnoea	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Hypoxia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Laryngeal stenosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Respiratory distress	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Vascular disorders Vena cava thrombosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.

Other adverse events

Measure	Alglucosidase Alfa n=4 participants at risk Alglucosidase alfa (Myozyme®) 20 mg/kg IV infusion qow (or optionally 20 mg/kg IV infusion every week \[qw\]) beginning from Day 0 to a minimum of 18 months or if the patient was \<6 months of age at the time of enrollment, until the patient was 2 years of age, along with methotrexate 0.4 mg/kg subcutaneously for 3 consecutive days qow beginning from Day 0 to Week 6 (9 doses) and rituximab 375 mg/m\^2 (or 12.5 mg/kg for patients with body surface area less than or equal to 0.5 m\^2) IV infusion qw beginning from Day -1 to Week 4 (4 doses) as per local prescribing information. An additional 4-week cycle of rituximab (up to 4 additional doses) and 6-week cycle of methotrexate (up to 9 additional doses) may have been administered within the first 6 months of the study as per local prescribing information.
Investigations Weight decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations White blood cell count decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations White blood cell count increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Metabolism and nutrition disorders Dehydration	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Metabolism and nutrition disorders Vitamin D deficiency	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Musculoskeletal and connective tissue disorders Muscle contracture	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Musculoskeletal and connective tissue disorders Muscle spasms	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Blood and lymphatic system disorders Anaemia	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Blood and lymphatic system disorders Lymphadenopathy	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Blood and lymphatic system disorders Neutropenia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Bradycardia	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Cyanosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Extrasystoles	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Cardiac disorders Tachycardia	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Ear and labyrinth disorders Tympanic membrane disorder	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Eye disorders Conjunctivitis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Eye disorders Eyelid ptosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Eye disorders Periorbital oedema	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Anal sphincter atony	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Anorectal disorder	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Constipation	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Diarrhoea	75.0% 3/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Duodenogastric reflux	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Flatulence	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Gastritis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Haematemesis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Haematochezia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Retching	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Teething	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Gastrointestinal disorders Vomiting	75.0% 3/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Catheter site discharge	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Catheter site erosion	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Catheter site erythema	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Catheter site pruritus	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Catheter site rash	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Catheter site related reaction	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Device dislocation	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Device occlusion	75.0% 3/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Face oedema	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Fatigue	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Intentional medical device removal by patient	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Medical device complication	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Oedema peripheral	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
General disorders Pyrexia	100.0% 4/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Body tinea	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Candidiasis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Catheter site cellulitis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Clostridium difficile colitis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Gastroenteritis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Impetigo	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Oral candidiasis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Otitis media	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Otitis media acute	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Sepsis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Serratia infection	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Tracheitis	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Urinary tract infection enterococcal	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Infections and infestations Viral infection	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Burns second degree	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Laceration	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Lip injury	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Medication error	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Overdose	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Procedural pain	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Procedural site reaction	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Spinal compression fracture	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Injury, poisoning and procedural complications Torus fracture	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Alanine aminotransferase increased	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Antibiotic resistant Staphylococcus test positive	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Aspartate aminotransferase increased	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Band neutrophil percentage increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood chloride decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood creatine phosphokinase MB increased	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood creatine phosphokinase increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood culture positive	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood immunoglobulin G increased	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood iron decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood lactate dehydrogenase increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood potassium decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood potassium increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood pressure increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Blood sodium decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Body temperature increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Clostridium test positive	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Eosinophil percentage increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Heart rate decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Heart rate irregular	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Lymphocyte percentage decreased	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Moraxella test positive	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Neutrophil count decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Neutrophil percentage increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Occult blood positive	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Oxygen saturation decreased	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Protein total decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Respiratory rate decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Respiratory rate increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Reticulocyte percentage increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Specific gravity urine increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Urinary hexose tetrasaccharide increased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Investigations Urine output decreased	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Musculoskeletal and connective tissue disorders Osteopenia	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Fibroma	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Nervous system disorders Clonus	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Nervous system disorders Tremor	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Psychiatric disorders Agitation	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Aspiration	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Atelectasis	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Bronchial secretion retention	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Bronchospasm	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Cough	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Diaphragm muscle weakness	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Diaphragmatic disorder	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Dyspnoea	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Increased bronchial secretion	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Laryngeal stenosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Pneumothorax	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Respiratory distress	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Tachypnoea	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Respiratory, thoracic and mediastinal disorders Wheezing	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Dermatitis contact	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Dermatitis diaper	75.0% 3/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Drug eruption	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Eczema	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Erythema	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Excessive granulation tissue	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Hair colour changes	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Hyperhidrosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Macule	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysaesthesia syndrome	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Papule	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Rash	75.0% 3/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Rash erythematous	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Rash macular	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Rash papular	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Red man syndrome	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Skin exfoliation	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Skin hypopigmentation	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Skin irritation	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Skin ulcer	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Skin and subcutaneous tissue disorders Swelling face	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Surgical and medical procedures Gastrointestinal tube removal	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Surgical and medical procedures Post procedural drainage	50.0% 2/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Vascular disorders Flushing	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Vascular disorders Hypotension	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Vascular disorders Pallor	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.
Vascular disorders Vena cava thrombosis	25.0% 1/4 • First dose of study drug up to end of study (up to Week 79) In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all patients who received at least 1 dose of alglucosidase alfa. AEs are listed independent of relationship to treatment.

Additional Information

Trial Transparency Team

Sanofi

Email: Contact-us@sanofi.com

Results disclosure agreements

• Principal investigator is a sponsor employee If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
• Publication restrictions are in place

Restriction type: OTHER