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Terminated Phase 3 Interventional Results available

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

ClinicalTrials.gov ID: NCT03783923

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.

ClinicalTrials.gov public records Last synced Oct 26, 2026, 7:02 AM EDT

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)

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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).

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

Interventions

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 31, 2019 (Actual)
Primary completion
Jan 1, 2021 (Actual)
Completion
Jan 1, 2021 (Actual)
Last update posted
Jun 27, 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 27, 2022 · Synced Oct 26, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT03783923 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →