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New hope for duchenne kids: drug combo after gene therapy tested

NCT ID NCT06100887

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 EDG-5506 (sevasemten) in 43 children and teens aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle damage markers. Participants are randomly assigned to receive the drug or a placebo, and later everyone can receive the drug.

Why investors are watching

Edgewise Therapeutics is testing EDG-5506 in children with Duchenne muscular dystrophy who have already had gene therapy. This small company's value depends on showing the drug is safe and works in this specific group, because many patients now receive gene therapy first. A positive readout would expand the drug's potential use, while a negative one would hurt the company's prospects.

If it works: If the trial shows EDG-5506 is safe and improves muscle biomarkers in these children, Edgewise could position the drug as a follow-on treatment for gene therapy patients. That would open a new market and strengthen the company's pipeline.

If it fails: The trial could fail to show benefit or reveal safety problems, which would set back the drug's development. Trials in this disease often fail, and a negative result would likely reduce the company's value and force it to rethink its strategy.

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

43 people

The number who actually took part.

Started

Mar 2024

Expected to finish

Mar 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

6 to 17 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 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. Key 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%. * Receipt of an investigational drug (other than the AAV-based gene therapy per Inclusion criteria) within 30 days or 5 half-lives (whichever is longer) of the Screening visit in the present study. * Receipt of an exon-skipping therapy within 6 months prior to the Screening 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

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rare Disease Research

    Hillsborough, North Carolina, 27278, 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 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

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