Status:
ACTIVE_NOT_RECRUITING
Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy
Lead Sponsor:
Edgewise Therapeutics, Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
6-17 years
Phase:
PHASE2
Brief Summary
The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy incl...
Detailed Description
FOX is a 2-part, multi-center, Phase 2 study to evaluate the effect of sevasemten (EDG-5506) on safety, pharmacokinetics and biomarkers of muscle damage in approximately 48 children and adolescents wi...
Eligibility Criteria
Inclusion
- Key
- 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).
- 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.
- Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.
- Key
Exclusion
- 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.
Key Trial Info
Start Date :
March 22 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2027
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT06100887
Start Date
March 22 2024
End Date
March 1 2027
Last Update
November 6 2025
Active Locations (7)
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1
UCLA Medical Center
Los Angeles, California, United States, 90095
2
UC Davis Medical Center
Sacramento, California, United States, 95817
3
University of Florida
Gainesville, Florida, United States, 32610
4
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States, 01605