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FSHD drug trial halted midway: what happened?

NCT ID NCT05397470

Disease control Sponsor: Fulcrum Therapeutics 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
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 tested a drug called losmapimod for people with a rare muscle-weakening disease called FSHD. The goal was to see if the drug could slow muscle loss and improve arm function over 48 weeks. About 260 adults with FSHD were randomly assigned to receive either losmapimod or a placebo. The study was terminated early, so results are limited.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

260 people

The number who actually took part.

Started

Jun 2022

Finished

Nov 2024

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

18 to 65 years

Sex

Anyone

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: * Participants must be between 18 and 65 years of age, inclusive. * Genetically confirmed diagnosis of FSHD 1 or FSHD 2. * Clinical severity score of 2 to 4 (Ricci Score; Range 0-5), at screening. Participants who are wheelchair-dependent or dependent on walker or wheelchair for activities are not permitted to enroll in the study. * Screening total RSA (Q1-Q4) without weight in the dominant UE assessed by RWS ≥ 0.2 and ≤ 0.7. * No contraindications to MRI. Key Exclusion Criteria: * Previously diagnosed cancer that has not been in complete remission for at least 5 years. Localized carcinomas of the skin and carcinoma in situ of the cervix that have been resected or ablated for cure are not exclusionary. * Participants who are on drug(s) or supplements that may affect muscle function, as determined by the Investigator: participants must be on a stable dose of that drug(s) or supplement for at least 3 months prior to the first dose of study drug and remain on that stable dose for the duration of the study. * Known active opportunistic or life-threatening infections including Human Immunodeficiency virus (HIV) and hepatitis B or C. * Known active or inactive tuberculosis infection. * Acute or chronic history of liver disease. * Known severe renal impairment. * History of cardiac dysrhythmias requiring anti-arrhythmia treatment(s); or history or evidence of abnormal ECGs. * Use of another investigational product within 30 days or 5 half-lives (whichever is longer) or currently participating in a study of an investigational device. * Current or anticipated participation in a natural history study. Previous participation is allowed but participants cannot continue after enrollment in Study 1821-FSH-301. * Known hypersensitivity to losmapimod or any of its excipients. * Previous participation in a Fulcrum-sponsored FSHD losmapimod study (FIS-001-2019 or FIS-002-2019). Note that all other inclusion and exclusion criteria are listed in the protocol and only key are presented.

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Conditions

The condition(s) this trial relates to.

facioscapulohumeral muscular dystrophy facioscapulohumeral muscular dystrophy 1 facioscapulohumeral muscular dystrophy 2 muscular dystrophy musculoskeletal system disorder neuromuscular disease

As listed by the trial registrant

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

Facioscapulohumeral muscular dystrophy (FSHD)

Contacts and locations

Locations

  • Aarhus Universitetshospital

    Aarhus, 8200, Denmark

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Fondazione Serena Onlus- Centro Clinico NEMO

    Milan, Lombardy, 20162, Italy

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Donostia

    San Sebastián, Guipuzkoa, 20014, Spain

  • Hospital Universitario Vall d'Hebron

    Barcelona, 08035, Spain

  • Institute de Myologie, Groupe Hospitalier Pitié-Salpêtrière

    Paris, 75013, France

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • LMU Klinikum Ludwig-Maximilians-Universität München

    München, 80336, Germany

  • Leiden University Medical Centre

    Leiden, Southern Holland, 2333 ZA, Netherlands

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Montreal Neurological Institute and Hospital

    Montreal, Quebec, H3A 2B4, Canada

  • Newcastle upon Tyne NHS Foundation Trust

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

  • Nice University Hospital - CHU Nice

    Nice, PACA, 06001, France

  • Ohio State University Medical Center

    Columbus, Ohio, 43210, United States

  • Radboudumc

    Nijmegen, Gelderland, 9101, Netherlands

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • The Ottawa Hospital Research Institute

    Ottawa, Ontario, K1Y 4E9, Canada

  • University College of London Hospitals

    London, WC1N 3BG, United Kingdom

  • University Hospital Bonn

    Bonn, 53127, Germany

  • University of Calgary

    Calgary, Alberta, T2N 4Z6, Canada

  • University of California Irvine

    Irvine, California, 92868, United States

  • University of California Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01655, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • University of Washington Medical Center

    Seattle, Washington, 98195, United States

  • Universitätsklinikum Ulm

    Ulm, 89081, Germany

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, 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.