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Hope for FSHD: new drug enters final stage of testing

NCT ID NCT07038200

Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 Jul 28, 2026 · Updated 2 times

Summary

This phase 3 trial tests an experimental drug called AOC 1020 in 200 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo by IV infusion. The study measures muscle strength, walking speed, and patient-reported physical function over time.

What this could mean

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

Active substance
AOC 1020 (del-brax)
What this could lead to
If successful, this could lead to the first approved treatment that slows or stops muscle weakness in people with FSHD, a rare genetic disease.
What could go wrong
This is a phase 3 trial, but it's still experimental. The drug may not improve muscle function enough, or could cause side effects. Results may not apply to all FSHD patients.

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

About 200 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2025

Expected to finish

Jul 2028

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

16 to 70 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.

Inclusion Criteria: * Clinical and genetic diagnosis of FSHD1 or FSHD2 * Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening * Adequate muscle strength based on QMT composite score Exclusion Criteria: * Breastfeeding, pregnancy, or intent to become pregnant during the study * Unwilling or unable to comply with contraceptive requirements * Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study * Blood Pressure \> 140/90 mmHg at Screening * Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer * Treatment with an oligonucleotide within 9 months of Screening

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Conditions

The condition(s) this trial relates to.

facioscapulohumeral muscular dystrophy facioscapulohumeral muscular dystrophy 1 facioscapulohumeral muscular dystrophy 2

As listed by the trial registrant

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

Facio-scapulo-humeral dystrophy Facioscapulohumeral dystrophy Facioscapulohumeral muscular dystrophy Facioscapulohumeral muscular dystrophy 1 Facioscapulohumeral muscular dystrophy 2 Facioscapulohumeral muscular dystrophy type 1 (FSHD1) Fascioscapulohumeral muscular dystrophy Fascioscapulohumeral muscular dystrophy type 1 Fascioscapulohumeral muscular dystrophy type 2 FSH muscular dystrophy FSHD FSHD - facioscapulohumeral muscular dystrophy FSHD1 FSHD2 Landouzy dejerine dystrophy Landouzy-dejerine muscular dystrophy Landouzy-dejerine syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    32 sites in 9 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • AP-HP Hopital Pitie-Salpetriere

    RECRUITING

    Paris, France

  • Aarhus University Hospital

    NOT_YET_RECRUITING

    Aarhus, Denmark

  • Academisch Ziekenhuis Leiden

    RECRUITING

    Leiden, Netherlands

  • Azienda Ospedaliero Universitaria Pisana

    RECRUITING

    Pisa, Italy

  • Azienda Ospedaliero-Universitaria Sant'Andrea

    RECRUITING

    Roma, Italy

  • CHU de Montpellier - Hopital Gui de Chauliac

    NOT_YET_RECRUITING

    Montpellier, France

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

    NOT_YET_RECRUITING

    Nice, France

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

    NOT_YET_RECRUITING

    Marseille, France

  • Duke University

    ACTIVE_NOT_RECRUITING

    Durham, North Carolina, 27708, United States

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

    RECRUITING

    Roma, Italy

  • Fondazione Serena ETS - Centro Clinico NeMO Milano

    RECRUITING

    Milan, Italy

  • Genge Partners Inc

    RECRUITING

    Montreal, Quebec, Canada

  • Georg-August-Universitaet Goettingen Stiftung oeffentlichen Rechts Universitaetsmedizin Goettingen

    RECRUITING

    Göttingen, Germany

  • Hospital Universitari de Bellvitge

    RECRUITING

    Barcelona, Spain

  • Hospital Universitari i Politecnic La Fe

    RECRUITING

    Valencia, Spain

  • Hospital Universitario Donostia

    RECRUITING

    San Sebastián, Spain

  • Hospital Universitario Vall d'Hebron

    RECRUITING

    Barcelona, Spain

  • Kansas University Medical Center

    ACTIVE_NOT_RECRUITING

    Kansas City, Kansas, 66205, United States

  • Kennedy Krieger Institute

    ACTIVE_NOT_RECRUITING

    Baltimore, Maryland, 21205, United States

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    RECRUITING

    München, Germany

  • National Center of Neurology and Psychiatry

    RECRUITING

    Tokyo, Japan

  • National Hospital Organization Osaka Toneyama Medical Center

    RECRUITING

    Osaka, Japan

  • National Hospital Organization Sendai Nishitaga Hospital

    RECRUITING

    Sendai, Japan

  • National Hospital for Neurology & Neurosurgery

    RECRUITING

    London, United Kingdom

  • Ohio State University

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43221, United States

  • Rigshospitalet

    RECRUITING

    Copenhagen, Denmark

  • Royal Hallamshire Hospital

    RECRUITING

    Sheffield, United Kingdom

  • Royal Victoria Infirmary

    RECRUITING

    Newcastle upon Tyne, United Kingdom

  • St. George's University Hospitals NHS Foundation Trust

    RECRUITING

    London, United Kingdom

  • Stanford University

    ACTIVE_NOT_RECRUITING

    Palo Alto, California, 94304, United States

  • Stichting Radboud Universitair Medisch Centrum

    RECRUITING

    Nijmegen, Netherlands

  • The Ottawa Hospital

    RECRUITING

    Ottawa, Ontario, Canada

  • The University of Osaka Hospital

    RECRUITING

    Osaka, Japan

  • Universitaetsklinikum Bonn

    RECRUITING

    Bonn, Germany

  • Universitaetsklinikum Ulm

    RECRUITING

    Ulm, Germany

  • University of Alberta Hospital

    RECRUITING

    Edmonton, Alberta, Canada

  • University of Calgary - Cumming School of Medicine

    RECRUITING

    Calgary, Alberta, Canada

  • University of California Irvine

    ACTIVE_NOT_RECRUITING

    Orange, California, 92868, United States

  • University of Colorado

    ACTIVE_NOT_RECRUITING

    Denver, Colorado, 80045, United States

  • University of Florida

    ACTIVE_NOT_RECRUITING

    Gainesville, Florida, 32608, United States

  • University of Iowa

    ACTIVE_NOT_RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Massachusetts

    ACTIVE_NOT_RECRUITING

    Worcester, Massachusetts, 01655, United States

  • University of Pennsylvania

    ACTIVE_NOT_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester Medical Center

    ACTIVE_NOT_RECRUITING

    Rochester, New York, 14642, United States

  • University of Texas Health Science Center at San Antonio

    ACTIVE_NOT_RECRUITING

    San Antonio, Texas, 78229, United States

  • Virginia Commonwealth University

    ACTIVE_NOT_RECRUITING

    Richmond, Virginia, 23298, United States

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