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Clinical Trials in the USA / NCT06100887
Active, not recruiting Phase 2

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

NCT06100887 · tracked via the Priya Life Science USA tracker
Sponsor
Edgewise Therapeutics, Inc.
Phase
Phase 2
Started
2024-03-22
Last updated
2025-11-06

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

Sevasemten Dose 1Sevasemten Dose 2Sevasemten Dose 3Placebo

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)

Trial sites (7)

FacilityCityRegionStatus
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

Other trials for the same condition

Official registry record

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.

View NCT06100887 on ClinicalTrials.gov ↗ ← All trials in the USA