NCT06666413

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 13 (estimated)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

Summary

This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Study details include: * The study duration: total study duration is approximately 64 weeks. * Screening period of up to 8 weeks * Treatment period of 52 weeks * Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks) * The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.

Conditions

• Glycogen Storage Disease Type II
• Pompe's Disease

Interventions

Type	Name	Description
DRUG	Avalglucosidase alfa	Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion

Timeline

• Start date: 2025-05-07
• Primary completion: 2028-05-02
• Completion: 2028-05-02
• First posted: 2024-10-30
• Last updated: 2026-03-23

Locations

1 site across 1 country: China