Open Actively Recruiting

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


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 including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.

Primary Purpose
Study Type
Phase 2


Healthy Volunteers
Minimum Age
6 Years
Maximum Age
14 Years

Key Inclusion Criteria:

  • Aged 6 to 14 with a documented mutation on the DMD gene and phenotype consistent with DMD.
  • Prior receipt of an AAV-based gene therapy (≥ 2 years after study drug administration in an open-label study 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 ≥ 20 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.

Join this Trial

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Study Stats
Protocol No.
Genetic and Rare Diseases
Emilie Douine-Barthelemy
  • UCLA Westwood
For Providers
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