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New drug trial hopes to slow muscle damage in kids with DMD

NCT ID NCT05540860

Disease control Sponsor: Edgewise Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental drug called sevasemten in 76 children aged 4-9 with Duchenne muscular dystrophy. The goal is to check the drug's safety, how the body processes it, and its effect on muscle health markers. Participants are randomly assigned to receive the drug or a placebo in the first part, then everyone gets the drug in the second part.

Why investors are watching

Edgewise Therapeutics is running a Phase 2 trial of an experimental drug in children with Duchenne muscular dystrophy, a serious muscle-wasting disease. The trial has two parts: a blinded, placebo-controlled section followed by an open-label section, and it will measure safety, drug levels, and biomarkers in 76 children. For a small company like Edgewise, this readout is a key test of whether the drug works and is safe enough to move forward, which could shape the company's entire value.

If it works: If the trial shows the drug is safe and moves disease-related biomarkers in a favorable direction, Edgewise could advance the drug to later-stage testing. That progress could attract partnership interest or support further development on its own.

If it fails: The trial could fail to show a meaningful effect on biomarkers, or safety problems could emerge, which would likely stall the drug's development. Clinical trials in this disease often fail, so a negative result is a real possibility that would hurt the company's prospects.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

76 people

The number who actually took part.

Started

Oct 2022

Expected to finish

Jan 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 9 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Common Inclusion Criteria: 1. A documented mutation on the DMD gene and phenotype consistent with Duchenne muscular dystrophy. 2. Able to complete the stand from supine in ≤ 10 seconds and able to perform the 4-stair climb in \< 10 seconds at the Screening visit. 3. Body weight greater than or equal to 15 kg at the Screening visit. For Cohorts 1, 2, 3, 4 and 5: Aged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit. For Cohort 2 Non-Steroid (Cohort 2NS): Aged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit. Key Common Exclusion Criteria: 1. 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 testing. 2. A forced vital capacity \< 60% predicted at the Screening visit for those participants who are \> 8 years old at Screening. 3. A cardiac echocardiography showing left ventricular ejection \< 45% at the Screening visit. 4. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study. 5. Receipt of a stable dose of an approved exon-skipping therapy with a treatment duration of less than 1 year prior to the Screening visit. For Cohort 2 Non-Steroid (Cohort 2NS): Receipt of oral corticosteroids for the treatment of Duchenne muscular dystrophy in the previous 6 months. Participants will not be tapered off steroids for the purpose of this study and oral corticosteroids for the treatment of Duchenne muscular dystrophy may be initiated after the Week 16 visit.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

Contacts and locations

Locations

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01605, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.