Health Condition 1: E740- Glycogen storage disease Health Condition 2: null- Pompe disease
Conditions
Interventions
Intervention1: alglucosidase alfa (4000 L material): Intravenous (IV) infusions of alglucosidase alfa (4000 L material) at a dose of 20 mg/kg of body weight every other week (qow)for 52 wk
Control Int
Sponsors
SanofisynthelaboIndia limited
Eligibility
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in cardiac function as measured by the left ventricular mass Z-score(LVM-Z)Timepoint: 52 wk | — |
Secondary
| Measure | Time frame |
|---|---|
| Estimated probability of survival Probability of invasive ventilator-free survival Change in motor development status as assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores Number of Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs)Timepoint: 52 wk | — |
Countries
Argentina, Brazil, Canada, China, Colombia, France, Germany, Italy, Poland, Russian Federation, Saudi Arabia, Turkey, United Kingdom, United States of America
Contacts
Public ContactSalini James
Sanofi
Outcome results
None listed