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EDG-5506 for Duchenne Muscular Dystrophy

Phase 2
Recruiting
Research Sponsored by Edgewise Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Body weight ≥ 20 kg at the Screening visit
Aged 6 to 14 with a documented mutation on the DMD gene and phenotype consistent with DMD
Must not have
Receipt of an exon-skipping therapy within 6 months prior to the Screening visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months

Summary

This trial tests a gene therapy in kids with Duchenne muscular dystrophy to see if it is safe and works. Part A is a blind placebo-controlled study, followed by an open-label study in Part B.

Who is the study for?
This trial is for children and adolescents aged 6 to 14 with Duchenne muscular dystrophy who have had gene therapy. They must be able to stand from lying down in under 8 seconds, climb stairs in less than 10 seconds, weigh at least 20 kg, and have been on a stable dose of corticosteroids for over six months.
What is being tested?
The FOX study tests EDG-5506's safety and effects compared to a placebo in kids previously treated with gene therapy for Duchenne muscular dystrophy. Part A is blinded and randomized; participants don't know if they're getting the drug or placebo. Part B will be open-label.
What are the potential side effects?
Specific side effects of EDG-5506 are not listed but may include reactions typical of clinical trials such as nausea, headache, fatigue or allergic reactions. The trial aims to assess these potential side effects thoroughly.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I weigh at least 20 kg.
Select...
I am between 6 and 14 years old with a confirmed DMD gene mutation.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have not taken exon-skipping therapy in the last 6 months.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of adverse events during treatment with sevasemten or placebo
Severity of adverse events during treatment with sevasemten or placebo
Secondary study objectives
Change from Baseline in fast skeletal muscle troponin I
Change from Baseline in serum creatine kinase
Incidence of abnormal clinical chemistry test results
+4 more

Trial Design

3Treatment groups
Experimental Treatment
Group I: Cohort 3Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Group II: Cohort 2Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Group III: Cohort 1Experimental Treatment2 Interventions
Drug: Sevasemten Drug: Placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Find a Location

Who is running the clinical trial?

Edgewise Therapeutics, Inc.Lead Sponsor
11 Previous Clinical Trials
861 Total Patients Enrolled
Sam Collins, MBBS, PhDStudy ChairEdgewise Therapeutics, Inc.
6 Previous Clinical Trials
479 Total Patients Enrolled
~27 spots leftby Oct 2025
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