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New hope for FSHD: experimental drug AOC 1020 completes early testing

NCT ID NCT05747924

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jul 17, 2026 · Updated 2 times

Summary

This study tested a new medicine called AOC 1020 in 90 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. The goal was to check if the drug is safe and how the body processes it. Participants received either the drug or a placebo by IV, and researchers measured side effects and signs of muscle improvement.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

90 people

The number who actually took part.

Started

Apr 2023

Finished

Mar 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

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: * FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor) * Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces) * At least 1 muscle region suitable for biopsy (testing provided by Sponsor) * Muscle weakness in both upper and lower body, as determined by Investigator Exclusion Criteria: * Pregnant or intends to become pregnant while on study, or active breastfeeding * Unwilling or unable to continue to comply with contraceptive requirements * Body mass index (BMI) \>35.0 kg/m2 at Screening * History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study * History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy * Anticipated survival less than 2 years * Blood or plasma donation within 16 weeks of Study Day 1 * Any contraindication to MRI * Any abnormal lab values, conditions or diseases that, in the opinion of the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study * Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) 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 frontometaphyseal dysplasia frontometaphyseal dysplasia 2 muscular dystrophy progressive muscular dystrophy

As listed by the trial registrant

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

Atrophies, facioscapulohumeral Atrophy, facioscapulohumeral Dystrophies, facioscapulohumeral muscular Dystrophies, landouzy-dejerine Dystrophy, facioscapulohumeral muscular Dystrophy, landouzy-dejerine Facio-scapulo-humeral dystrophy Facioscapulohumeral atrophy Facioscapulohumeral muscular dystrophy 1 Facioscapulohumeral muscular dystrophy 2 Fascioscapulohumeral muscular dystrophy Fascioscapulohumeral muscular dystrophy type 1 Fascioscapulohumeral muscular dystrophy type 2 FMD FMD2 FSH FSH muscular dystrophy FSHD FSHD1 FSHD2 Landouzy dejerine dystrophy Landouzy-dejerine muscular dystrophy Landouzy-dejerine syndrome Muscular dystrophies Muscular dystrophy, facioscapulohumeral Muscular dystrophy, landouzy dejerine Progressive muscular dystrophy

Contacts and locations

Locations

  • Duke University

    Durham, North Carolina, 27708, United States

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • University College London

    London, WIT 7HA, United Kingdom

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of California San Diego

    San Diego, California, 92093, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Ottawa

    Ottawa, Ontario, K1Y 4E9, Canada

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Sheffield

    Sheffield, S10 2TN, United Kingdom

  • University of Texas Southwestern

    Dallas, Texas, 75390, United States

  • University of Washington

    Seattle, Washington, 98104, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

More trials for these conditions

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