NCT04910776

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

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)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 0 Days – 12 Months
• Healthy volunteers: Not accepted

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.

Conditions

• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
DRUG	avalglucosidase alfa	Sterile lyophilized powder intravenous (IV) infusion

Timeline

• Start date: 2021-09-01
• Primary completion: 2026-03-02
• Completion: 2027-08-10
• First posted: 2021-06-02
• Last updated: 2026-04-08

Locations

16 sites across 9 countries: United States, Belgium, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study