Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for rare muscle disease: experimental drug enters human trials

NCT ID NCT06131983

Disease control Sponsor: Sarepta Therapeutics, 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 12, 2026 · Updated 2 times

Summary

This study tests an experimental drug called ARO-DUX4 in people with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a genetic condition that causes muscle weakness. The main goal is to check the drug's safety and how the body processes it. About 60 adults and teens will receive either the drug or a placebo, with some receiving up to four doses. The study also looks at how the drug affects muscle tissue through biopsies and MRI scans.

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

About 60 people

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

Started

Jun 2024

Expected to finish

Dec 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.

Key Inclusion Criteria: * Genetically confirmed FSHD1 based on screening evaluation or source verifiable medical record * Clinical severity score between 3 and 8 (scale, 0 to 10) * Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with muscle fat fraction ≥10% and less than approximately 40% * Males or nonpregnant, nonlactating females ≥18 years of age who do not plan to become pregnant during the study, with an upper age limit of ≤70 years * Able and willing to provide written informed consent prior to the performance of any study specific procedures * Participants with a body mass index (BMI) between 18.0 and 35.0 kilograms/square meter, inclusive. A participant with FSHD1 and a BMI outside this range may be allowed into the study at the discretion of the principal investigator. * Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader * A 12-lead electrocardiogram at screening with no abnormalities that may compromise participant's safety in the study * Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 9 months following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 9 months following the end of study or last dose of study medication, whichever is later. Key Exclusion Criteria: * Human immunodeficiency virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at screening * Seropositive for hepatitis B or hepatitis C at screening * Uncontrolled hypertension * Severe cardiovascular disease * History of thrombolic events * Platelet count less that the lower limit of normal at screening * History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives. * Any contraindication to muscle biopsy or MRI Note: additional inclusion/exclusion criteria may apply per protocol

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Facio-scapulo-humeral dystrophy are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

facioscapulohumeral muscular dystrophy facioscapulohumeral muscular dystrophy 1

As listed by the trial registrant

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

Facio-scapulo-humeral dystrophy Muscular dystrophy, facioscapulohumeral

How to take part

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

  1. The places running it

    16 sites in 7 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

  • Alfred Health

    RECRUITING

    Melbourne, Victoria, 3004, Australia

  • Centre For Human Drug Research

    RECRUITING

    Leiden, 2333, Netherlands

  • Centro Clinico Nemo Milann

    RECRUITING

    Milan, 20162, Italy

  • Fondazione Policlinico Universitario A. Gemelli

    RECRUITING

    Roma, 00168, Italy

  • Hospital Universitari l Politecnic La Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

  • Hospital Universitario Ramon y Cajal

    RECRUITING

    Madrid, 28034, Spain

  • Hospital Universitario Vall d'Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Liverpool Hospital

    RECRUITING

    Liverpool, New South Wales, 2170, Australia

  • McGill University

    RECRUITING

    Montreal, Quebec, H3A2B4, Canada

  • Neurologische Universitätsklinik Am Rku

    RECRUITING

    Ulm, 89081, Germany

  • New Zealand Clinical Research

    RECRUITING

    Auckland, 1010, New Zealand

  • Sunshine Coast University Hospital

    RECRUITING

    Birtinya, Queensland, 4575, Australia

  • University of Alberta

    RECRUITING

    Edmonton, Alberta, T6G2G4, Canada

  • University of Calgary

    NOT_YET_RECRUITING

    Calgary, Alberta, T2N4Z6, Canada

  • Universitätsklinikum München

    RECRUITING

    München, 80336, Germany

  • Wesley Research Institute

    RECRUITING

    Auchenflower, Queensland, 4066, Australia

More trials for these conditions

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