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)

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, but until avalglucosidase alfa is approved in the patient's country or up to 4.08 years, whichever comes first.


Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date: September 1, 2021
Estimated Primary Completion Date: December 27, 2024
Estimated Study Completion Date: January 1, 2027

Arm:
- Experimental: Avalglucosidase alfa

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