Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase Alfa

• Status: Recruiting
• Trial end date: 2026-12-01

Summary

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and female participants less than or equal to 6 months of age with IOPD.

Study details include:

• Study duration: Screening - up to 4 weeks;

• Primary Analysis Period (PAP) - 52 weeks;

• Extended Treatment Period (ETP) - 52 weeks;

• Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years.

• Treatment duration: Up to 4 years

• Visit frequency: every other week and potentially every week

• Phase: Phase 3

Details

• Lead Sponsor: Sanofi