A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Public ClinicalTrials.gov record NCT03783923. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Study identification
- NCT ID
- NCT03783923
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- PTC Therapeutics
- Industry
- Enrollment
- 11 participants
Conditions and interventions
Conditions
Interventions
- Deflazacort Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 30, 2019
- Primary completion
- Dec 31, 2020
- Completion
- Dec 31, 2020
- Last update posted
- Jun 26, 2022
2019 – 2021
United States locations
- U.S. sites
- 8
- U.S. states
- 8
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Rare Disease Research, LLC | Atlanta | Georgia | 30324 | — |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | — |
| The University of Kansas Medical Center | Kansas City | Kansas | 66160 | — |
| Hugo W Moser Research Institute at Kennedy Krieger Institute | Baltimore | Maryland | 21205 | — |
| University of Minnesota | Minneapolis | Minnesota | 55455 | — |
| Washington University School of Medicine | St Louis | Missouri | 63110 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
| University of Washington | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 10 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03783923, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 26, 2022 · Synced Jul 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03783923 live on ClinicalTrials.gov.