Hope for FSHD: new drug enters final stage of testing

NCT ID NCT07038200

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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.

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

AP-HP Hopital Pitie-Salpetriere
NOT_YET_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
NOT_YET_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
NOT_YET_RECRUITING

Göttingen, Germany
Hospital Universitari de Bellvitge
RECRUITING

Barcelona, Spain
Hospital Universitari i Politecnic La Fe
RECRUITING

Valencia, Spain
Hospital Universitario Donostia
NOT_YET_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
NOT_YET_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
NOT_YET_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