An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
Public ClinicalTrials.gov record NCT03917719. 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
An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD.
Study identification
- NCT ID
- NCT03917719
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 130 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 4 Years to 12 Years
- Sex
- Male
- 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
- Mar 14, 2019 (Actual)
- Primary completion
- Oct 26, 2020 (Actual)
- Completion
- Oct 26, 2020 (Actual)
- Last update posted
- Nov 23, 2020
2019 – 2020
United States locations
- U.S. sites
- 15
- U.S. states
- 13
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UC Davis | Sacramento | California | 95817 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | — |
| University of Iowa Children's Hospital | Iowa City | Iowa | 52242 | — |
| University of Kansas Medical Center | Fairway | Kansas | 66205 | — |
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | — |
| Johns Hopkins School of Medicine | Baltimore | Maryland | 21287 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| University of Michigan | Ann Arbor | Michigan | 48109 | — |
| Las Vegas Clinic | Las Vegas | Nevada | 89145 | — |
| Shriners Hospital for Children | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37212 | — |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | — |
| University of Texas Health Science Center at San Antonio | San Antonio | Texas | 78229 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
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 8 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03917719, 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 Nov 23, 2020 · Synced Sep 30, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03917719 live on ClinicalTrials.gov.