Trials / Terminated
TerminatedNCT03783923
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
- Status
- Terminated
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 11 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Deflazacort | Deflazacort tablet will be administered as per the dose and schedule specified in the arm. |
Timeline
- Start date
- 2019-10-31
- Primary completion
- 2021-01-01
- Completion
- 2021-01-01
- First posted
- 2018-12-21
- Last updated
- 2022-06-27
- Results posted
- 2022-06-27
Locations
18 sites across 8 countries: United States, Canada, Denmark, France, Germany, Norway, Russia, Sweden
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03783923. Inclusion in this directory is not an endorsement.