drugset / Trial / NCT04060199
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
Timeline
- Start
- 2020-04-14
- Primary completion
- 2023-10-19
- Completion
- 2023-10-19
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Viltolarsen | Unknown | 80 mg/kg | Intravenous |