EDG-5506 for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

+6 other locations

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Edgewise Therapeutics, Inc.

Must be taking: Corticosteroids

Must not be taking: Exon-skipping therapy

Disqualifiers: Low LVEF, others

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Do I have to stop taking my current medications for the trial?

The trial requires that participants have been on a stable dose of corticosteroids for at least 6 months before starting. It doesn't specify if you need to stop other medications, so it's best to discuss with the trial team.

Research Team

Sam Collins, MBBS, PhD

Principal Investigator

Edgewise Therapeutics, Inc.

Eligibility Criteria

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.

Inclusion Criteria

I received AAV-based gene therapy over 2 or 3 years ago, depending on the study type.

I can stand up from lying down in less than 8 seconds and climb 4 stairs in less than 10 seconds.

I weigh at least 20 kg.

Exclusion Criteria

Your heart's pumping ability is less than 40% as shown in a heart ultrasound during the screening visit.

I haven't taken any experimental drugs recently, except for the AAV-based gene therapy.

I have not taken exon-skipping therapy in the last 6 months.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Treatment (Part A)

Participants receive a 12-week randomized, double-blind, placebo-controlled treatment with sevasemten or placebo

12 weeks

Open-label Extension (Part B)

Participants continue treatment in a 40-week open-label extension period with sevasemten

40 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• EDG-5506 (Other)

Trial Overview 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.

Participant Groups

Group I: Cohort 3 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group II: Cohort 2 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Group III: Cohort 1 Experimental Treatment 2 Interventions

Drug: Sevasemten Drug: Placebo

Who Is Running the Clinical Trial?

Edgewise Therapeutics, Inc.

Lead Sponsor

Trials

13

Recruited

1,000+