Edgewise Sevasemten (EDG-5506) Phase 2 Clinical Trial for Gene Therapy-Treated Duchenne Patients (FOX)
Active, Not RecruitingPhase 2Improving Muscle Growth & Protection
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.
Clinical Trial Registry
NCT ID
NCT06100887This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.