Clinical study for treatment-naïve IOPD babies to evaluate efficacy and safety of ERT with avalglucosidase alfa (Baby-COMET)

Clinical study for treatment-naïve IOPD babies to evaluate efficacy and safety of ERT with avalglucosidase alfa (Baby-COMET)

ClinicalTrials.gov ID: NCT04910776
Sponsor: Sanofi
Information provided by: Sanofi (Responsible Party)
Last Update Posted: 2024-07-16

Brief Summary:
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants 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

Detailed Description:
Study duration may be variable by country, including at least completion of the PAP and ETP, and up to 4.08 years.

Official Title:
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)

Intervention / Treatment: 
- Drug: avalglucosidase alfa

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