NCT06100887

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

A Phase 2 Study to Evaluate the Effect of EDG-5506 on Safety, Pharmacokinetics, and Biomarkers in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Edgewise Therapeutics, Inc. · Industry
• Sex: Male
• Age: 6 Years – 17 Years
• Healthy volunteers: Not accepted

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.

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 with Duchenne muscular dystrophy treated with oral, once-daily sevasemten. This study will have up to a 4-week Screening period, a 12-week randomized double-blind, placebo-controlled treatment period (Part A), followed by up to a 144-week open-label extension period (Part B). Approximately forty-eight (48) participants aged 6 to 17, inclusive, will be randomized to sevasemten or placebo in a 2:1 ratio. Three dose cohorts (Cohort 1, Cohort 2 and Cohort 3) of approximately 12 participants each will be enrolled. Approximately 12 additional participants may be added to 1 of these cohorts. After review of emerging data, the protocol was amended so all dose cohorts receive the same dose in Part B.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	Sevasemten Dose 1	Sevasemten is administered orally once per day
DRUG	Sevasemten Dose 2	Sevasemten is administered orally once per day
DRUG	Sevasemten Dose 3	Sevasemten is administered orally once per day
DRUG	Placebo	Placebo is administered orally once per day

Timeline

• Start date: 2024-03-22
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2023-10-25
• Last updated: 2025-11-06

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study