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Myozyme in Infantile-Onset Pompe Disease
NCT00053573Phase 2
Key facts
- Status
- Completed
- Start
- Feb 2003
- Primary completion
- Jul 2006
- Countries
- 4
- Sponsor
- Sanofi
- Modality
- Replacement Enzymes
- Drugs
- alglucosidase alfa
- Conditions
- Pompe disease
01
Overview
Phase 2, open-label, single-arm trial of alglucosidase alfa in Pompe disease, sponsored by Sanofi. Primary endpoints: Safety of Myozyme at 52 weeks, Proportion of patients alive at 52 weeks and 2 more. Started Feb 2003; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
Experimental Arm
Myozyme 20-40 mg/kg qow
03
Who it enrols
Infants 6-36 months old with documented infantile-onset Pompe disease (GAA deficiency <= 2% and LVMI > 2 SD), without cardiac failure (LVEF >= 40%) or prior ERT.
04
Endpoints
Primary
Safety of Myozyme
Proportion of patients alive
Pharmacokinetic profile of Myozyme
Pharmacodynamic profile of Myozyme
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