drugset / Trial / NCT02420379
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Timeline
- Start
- 2015-06-30
- Primary completion
- 2018-12-17
- Completion
- 2018-12-17
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Eteplirsen | Antisense oligonucleotide | 30 mg/kg | Intravenous |