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

StatusRecruiting
PhasePhase 4
Started2025-05-07
View on ClinicalTrials.gov ↗
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.

Amendment record

Amended 3 times since 2024-10-29; most recent filed 2026-03-19. Every amendment is listed below.

Dates here are the day the sponsor filed the amendment, not the day ClinicalTrials.gov published it. Version history last read from ClinicalTrials.gov on 2026-08-30.

2026-03-19
Amendment Study Status, Study Description, Eligibility v3
2025-05-27
Amendment Not Yet RecruitingRecruiting Study Status, Contacts/Locations v2
2025-04-16
Amendment Study Status v1
2024-10-29
Original filing
Trial Details
NCT Number NCT06666413
Lead Sponsor Genzyme, a Sanofi Company
Conditions Glycogen Storage Disease Type II, Pompe's Disease
Enrollment 13 participants
Start Date 2025-05-07
Primary Completion 2028-05-02 (estimated)
Study Completion 2028-05-02 (estimated)
Updated on ClinicalTrials.gov 2026-03-23