Avalglucosidase alfa french post-trial access for participants with Pompe disease (PTA Avalglucosidase)
Avalglucosidase alfa french post-trial access for participants with Pompe disease (PTA Avalglucosidase)
ClinicalTrials.gov ID: NCT05164055
Sponsor: Genzyme, a Sanofi Company
Information provided by: Sanofi (Genzyme, a Sanofi Company) (Responsible Party)
Last Update Posted: 2023-09-28
Brief Summary:
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to enzyme replacement therapy (ERT) with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until December 2024, whichever comes first.µ
- Study visit frequency: every 2 weeks
Detailed Description:
Treatment duration approximately 2 years and 6 months: until reimbursement of avalglucosidase alfa in France or until December 2024, whichever comes first.
Official Title:
A French Multicenter Open Label Phase 4 Extension Study of Long-term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Alfa Development Studies in France
Intervention / Treatment:
- Drug: Avalglucosidase alfa (GZ402666)
| Category | Value |
|---|---|
| Study Start (Actual) |
2022-07-11
|
| Primary Completion (Estimated) |
2024-12-31
|
| Study Completion (Estimated) |
2024-12-31
|
| Enrollment (Actual) | 17 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
PTA17333
2021-002590-26 (EudraCT Number) U1111-1266-5434 (Registry Identifier) (REGISTRY: ICTRP) |