A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Phase 4

Terminated

Conditions: Pompe Disease (Infantile-Onset)
Glycogenosis 2
Acid Maltase Deficiency
Glycogen Storage Disease Type II (GSD II)

Brief Summary: A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.

Recruitment & Eligibility

Inclusion Criteria

The participant's parent/legal guardian was willing and able to provide signed informed consent.
The participant might be less than or equal to 12 months of age.
The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
The participant might be naïve to treatment with alglucosidase alfa.

Exclusion Criteria

The participant was cross-reactive immunologic material negative.
The participant required invasive ventilator support at the time of enrollment.
The participant had decompensated clinical heart failure.
The participant had a major congenital abnormality, excluding cardiac hypertrophy.
The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
The participant was currently receiving any investigational product.
The participant was participating in another clinical study.
The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.

Arm && Interventions

Group	Intervention	Description
Alglucosidase Alfa 4000 L material (Non-US participants)	alglucosidase alfa	Alglucosidase alfa 4000 L material for 52 weeks.
Alglucosidase Alfa 160 L material (US participants)	alglucosidase alfa	Alglucosidase alfa 160 L material for 52 weeks.

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Cardiac Function at Week 52	Baseline, Week 52	Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants With Estimated Probability of Survival	Up to Week 52
Number of Participants With Invasive Ventilator-Free Survival	Up to Week 52	Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported.
Change From Baseline in Motor Development Status at Week 52	Baseline, Week 52	Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. 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 was 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 was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions.