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)

• Registration Number: NCT01597596
• Lead Sponsor: Genzyme, a Sanofi Company

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-05-10
• Study First Submitted QC: 2012-05-10
• Study First Posted: 2012-05-14
• Study Start: 2012-08
• Primary Completion: 2014-12
• Study Completion: 2014-12
• Results First Submitted: 2015-09-25
• Status Verified: 2015-12
• Results First Submitted QC: 2015-12-14
• Last Update Submitted: 2015-12-14
• Results First Posted: 2016-01-18
• Last Update Posted: 2016-01-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 4

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

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
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.
Percentage of Participants With Estimated Probability of Survival	Up to Week 52