drugset / Trial / NCT03783923
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
NaSingle-groupOpen-labelTreatment
Summary
This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).
Timeline
- Start
- 2019-10-31
- Primary completion
- 2021-01-01
- Completion
- 2021-01-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Deflazacort | Other / unclassified | 0.6 mg/kg | Oral |