A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated
Phase 3
- Conditions
- Health Condition 1: E740- Glycogen storage diseaseHealth Condition 2: null- Pompe disease
- Registration Number
- CTRI/2015/04/005672
- Lead Sponsor
- SanofisynthelaboIndia limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Other (Terminated)
- Sex
- Not specified
- Target Recruitment
- 24
Inclusion Criteria
The patient must be less than or equal to 12 months of age; The patient must have documented GAA enzyme deficiency from blood, skin, or muscle tissue; The patient must be naïve to treatment with alglucosidase alfa
For complete list of inclusion criteria, please contact the sponsor
Exclusion Criteria
The patient is cross-reactive immunologic material negative; The patient requires invasive ventilator support at the time of enrollment;The patient has decompensated clinical heart failure;
For complete list of inclusion criteria, please contact the sponsor
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change from baseline in cardiac function as measured by the left ventricular mass Z-score(LVM-Z)Timepoint: 52 wk
- Secondary Outcome Measures
Name Time Method Estimated probability of survival <br/ ><br>Probability of invasive ventilator-free survival <br/ ><br>Change in motor development status as assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores <br/ ><br>Number of Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs)Timepoint: 52 wk