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 Recruiting→Recruiting Study Status, Contacts/Locations v2
2025-04-16
Amendment Study Status v1
2024-10-29
Original filing
| 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 |