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

NCT ID: NCT06100887

Last Updated: 2025-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-22

Study Completion Date

2027-03-31

Brief Summary

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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

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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

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Duchenne Muscular Dystrophy

Keywords

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Cohort 1

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 1

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 2

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 2

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Cohort 3

Drug: Sevasemten Drug: Placebo

Group Type EXPERIMENTAL

Sevasemten Dose 3

Intervention Type DRUG

Sevasemten is administered orally once per day

Placebo

Intervention Type DRUG

Placebo is administered orally once per day

Interventions

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Sevasemten Dose 1

Sevasemten is administered orally once per day

Intervention Type DRUG

Sevasemten Dose 2

Sevasemten is administered orally once per day

Intervention Type DRUG

Sevasemten Dose 3

Sevasemten is administered orally once per day

Intervention Type DRUG

Placebo

Placebo is administered orally once per day

Intervention Type DRUG

Eligibility Criteria

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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.

* Receipt of an exon-skipping therapy within 6 months prior to the Screening visit.

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%.
Minimum Eligible Age

6 Years

Maximum Eligible Age

17 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Edgewise Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCLA Medical Center

Los Angeles, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Rare Disease Research

Hillsborough, North Carolina, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Countries

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United States

Related Links

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http://edgewisetx.com

Sponsor Website

Other Identifiers

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EDG-5506-215

Identifier Type: -

Identifier Source: org_study_id