drugset / Trial / NCT01957059
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
Non-randomizedParallel-groupOpen-labelTreatment
Summary
The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.
Timeline
- Start
- 2013-06
- Primary completion
- 2016-08-03
- Completion
- 2016-08-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BMN053 | Unknown | 1 mg/kg | Intravenous |
| Subject | BMN053 | Unknown | 3 mg/kg | Intravenous |
| Subject | BMN053 | Unknown | 6 mg/kg | Intravenous |
| Subject | BMN053 | Unknown | 9 mg/kg | Intravenous |