Sevasemten Dose 1: Sevasemten is administered orally once per day
Sevasemten Dose 2: Sevasemten is administered orally once per day
Sevasemten Dose 3: Sevasemten is administered orally once per day
Placebo: Placebo is administered orally once per day
Study 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 including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.
Eligibility
Sex
MALE
Min age
6 Years
Max age
17 Years
Healthy volunteers
No
Key Inclusion Criteria:
* 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 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.
Primary outcome measure(s)
Number of adverse events during treatment with sevasemten or placebo — 36 months All participants
Severity of adverse events during treatment with sevasemten or placebo — 36 months All participants
Trial sites (7)
Facility
City
Region
Status
UCLA Medical Center
Los Angeles
California
UC Davis Medical Center
Sacramento
California
University of Florida
Gainesville
Florida
University of Massachusetts Memorial Medical Center
Worcester
Massachusetts
Washington University School of Medicine
St Louis
Missouri
Rare Disease Research
Hillsborough
North Carolina
Nationwide Children's Hospital
Columbus
Ohio
More Edgewise Therapeutics, Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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