New hope for rare muscle disease: experimental drug enters human trials
NCT ID NCT06131983
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Alfred Health
RECRUITINGMelbourne, Victoria, 3004, Australia
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Centre For Human Drug Research
RECRUITINGLeiden, 2333, Netherlands
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Centro Clinico Nemo Milann
RECRUITINGMilan, 20162, Italy
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Fondazione Policlinico Universitario A. Gemelli
RECRUITINGRoma, 00168, Italy
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Hospital Universitari l Politecnic La Fe de Valencia
RECRUITINGValencia, 46026, Spain
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Hospital Universitario Ramon y Cajal
RECRUITINGMadrid, 28034, Spain
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Hospital Universitario Vall d'Hebron
RECRUITINGBarcelona, 08035, Spain
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Liverpool Hospital
RECRUITINGLiverpool, New South Wales, 2170, Australia
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McGill University
RECRUITINGMontreal, Quebec, H3A2B4, Canada
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Neurologische Universitätsklinik Am Rku
RECRUITINGUlm, 89081, Germany
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New Zealand Clinical Research
RECRUITINGAuckland, 1010, New Zealand
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Sunshine Coast University Hospital
RECRUITINGBirtinya, Queensland, 4575, Australia
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University of Alberta
RECRUITINGEdmonton, Alberta, T6G2G4, Canada
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University of Calgary
NOT_YET_RECRUITINGCalgary, Alberta, T2N4Z6, Canada
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Universitätsklinikum München
RECRUITINGMünchen, 80336, Germany
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Wesley Research Institute
RECRUITINGAuchenflower, Queensland, 4066, Australia
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New hope for FSHD: Long-Term drug safety trial underway
- New hope for FSHD: experimental drug AOC 1020 completes early testing
- Inflammation may be key in rare muscle disease
- New study aims to sharpen tools for testing kids with rare muscle disease
- FSHD patients unite: new registry aims to accelerate research and care