drugset / Trial / NCT05429372
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
NaSingle-groupOpen-labelTreatment
Summary
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
Timeline
- Start
- 2022-08-08
- Primary completion
- 2025-10-03
- Completion
- 2025-10-03
Outcome
Outcome not reported
Stopped: “All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | fordadistrogene movaparvovec | Gene therapy (AAV / viral vector) | — | Intravenous |