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New pill aims to slow muscle damage in becker MD

NCT ID NCT05291091

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 21, 2026 · Updated 3 times

Summary

This phase 2 trial tests a daily oral drug called sevasemten (EDG-5506) in 244 adults and adolescents with Becker muscular dystrophy, a condition that causes progressive muscle weakness. The study compares the drug to a placebo to see if it can reduce muscle damage, improve walking ability, and be safe. Participants take the drug once daily for several months, and researchers track changes in muscle enzymes and physical function.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Sevasemten (EDG-5506), a daily oral drug
What this could lead to
If it works, this could slow muscle damage and improve daily function in people with Becker muscular dystrophy.
What could go wrong
This is a mid-stage trial with 244 participants, so results may not confirm benefit. Side effects are still being studied, and the drug may not work for everyone.

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

244 people

The number who actually took part.

Started

Nov 2022

Finished

Sep 2026

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

12 to 50 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.

GRAND CANYON eligibility is listed below. Key Inclusion Criteria: 1. Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy, and history of being ambulatory beyond 16 years of age without steroids; history of being ambulatory beyond 18 years of age with steroids. 2. Able to complete the 100-meter timed test in \< 200 seconds with or without use of mobility aid devices. 3. Able to perform the North Star Ambulatory Assessment scale and achieve a score of 5 to 32, inclusive. Key Exclusion Criteria: 1. Medical history or clinically significant physical examination/laboratory result that, in the opinion of the investigator, would render the participant unsuitable for the study. This includes contraindications to magnetic resonance imaging such as non-compatible implanted medical devices or severe claustrophobia. 2. Cardiac echocardiogram ejection fraction \< 40% 3. Forced vital capacity predicted \<60% or using daytime ventilatory support 4. Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months. 5. Receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) of the screening visit in the present study.

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Conditions

The condition(s) this trial relates to.

Becker muscular dystrophy Duchenne muscular dystrophy

As listed by the trial registrant

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

Becker muscular dystrophy

Contacts and locations

Locations

  • AP-HP Hopital Pitie-Salpetriere

    Paris, 75013, France

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Azienda Ospedale - Università Padova

    Padova, 35128, Italy

  • CHU de Nantes

    Nantes, 44093, France

  • CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires

    Nice, 06001, France

  • Centre Hospitalier Régional de la Citadelle

    Liège, 4000, Belgium

  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

    Marseille, 13005, France

  • Fondazione IRCCS Ca'Granda Ospedale Maggiore Policlinico di Milano

    Milan, 20122, Italy

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    Rome, 00168, Italy

  • Hadassah University Hospital

    Jerusalem, 91240, Israel

  • Hospital Universitari de Bellvitge

    Barcelona, 08907, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Donostia

    Donostia / San Sebastian, 20014, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035, Spain

  • Indiana University School of Medicine

    Indianapolis, Indiana, 46202, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    Munich, 80336, Germany

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZA, Netherlands

  • National Neuromuscular Research Institute

    Austin, Texas, 78759, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Neurology Rare Disease Center

    Denton, Texas, 76208, United States

  • Newcastle Freeman Hospital

    Newcastle, NE7 7DN, United Kingdom

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • Optimal Clinical Trials

    Auckland, 1010, New Zealand

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Rare Disease Research, LLC NC

    Hillsborough, North Carolina, 27278, United States

  • Rigshospitalet

    Copenhagen, Denmark

  • Salford Royal Hospital

    Salford, M68HD, United Kingdom

  • Schneider Children's Hospital of Israel

    Petah Tikva, 49202, Israel

  • St Vincent's Hospital Melbourne

    Fitzroy, VIC, 3065, Australia

  • St. George's University Hospitals NHS Foundation Trust

    London, SW17 0QT, United Kingdom

  • Stanford Neuroscience Health Center

    Palo Alto, California, 94304, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • UC Denver

    Aurora, Colorado, 80045, United States

  • UC Irvine Medical Center

    Orange, California, 92868, United States

  • UC San Diego

    La Jolla, California, 92037, United States

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • Universitaire Ziekenhuizen Leuven

    Leuven, Belgium

  • University College London Hospital

    London, NW1 2PG, United Kingdom

  • University Hospital Gent

    Ghent, Belgium, 9000, Belgium

  • University of Cincinnati Gardner Neuroscience Institute

    Cincinnati, Ohio, 45219, United States

  • University of Florida

    Gainesville, Florida, 32611, 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

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • Virginia Commonwealth University Health

    Richmond, Virginia, 23298, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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