Edgewise Sevasemten (EDG-5506) Phase 2 Clinical Trial for Gene Therapy-Treated Duchenne Patients (FOX)
Study Overview
- Age
- 6–17 years
- Phase
- Phase 2
- Sponsor
- Edgewise Therapeutics
- Therapeutic Approach
- Improving Muscle Growth & Protection
- Variant Requirement
- Aged 6 to 17 with a documented mutation on the DMD gene and phenotype consistent with DMD. Prior receipt of an AAV-based gene therapy (≥ 2 years after documented receipt of gene therapy administration or ≥ 3 years after randomization in a randomized study).
- Eligible Sex
- Male
- Ambulation
- Ambulatory
- Study Start (Actual)
- 2024-03-22
- Primary Completion (Estimated)
- 2027-03
- Study Completion (Estimated)
- 2027-03
- Enrollment (Estimated)
- 43
- Countries
- United States
Study Requirements and Criteria
Steroid Use
Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.
Inclusion Criteria
- Able to complete stand from supine in ≤ 8 seconds at the Screening visit and able to perform the 4-stair climb in < 10 seconds at the Screening visit.
- Body weight ≥ 15 kg at the Screening visit.
Exclusion Criteria
- Medical history or clinically significant physical exam/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes venous access that would be too difficult to facilitate repeated blood sampling.
- Screening visit cardiac echocardiography showing left ventricular ejection fraction (LVEF) < 40%.
- Receipt of an investigational drug (other than the AAV-based gene therapy per Inclusion criteria) within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study.
- Receipt of an exon-skipping therapy within 6 months prior to the Screening visit.
Contacts and Locations
Contacts and Locations
Contact
Not yet provided
Locations
This study has 7 locations
United States
California Locations
Los Angeles, California, United States, 90095
UCLA Medical Center
Sacramento, California, United States, 95817
UC Davis Medical Center
Florida Locations
Gainesville, Florida, United States, 32610
University of Florida
Massachusetts Locations
Worcester, Massachusetts, United States, 01605
University of Massachusetts Memorial Medical Center
Missouri Locations
St Louis, Missouri, United States, 63110
Washington University School of Medicine
North Carolina Locations
Hillsborough, North Carolina, United States, 27278
Rare Disease Research
Ohio Locations
Columbus, Ohio, United States, 43205
Nationwide Children's Hospital
Clinical Trial Registry
NCT ID
NCT06100887This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.