New hope for rare muscle disease: experimental drug enters human trials
NCT ID NCT06131983
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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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the original study
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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.
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.
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
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Research Site
WITHDRAWNBangkok, 10700, Thailand
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Research Site 1
RECRUITINGBirtinya, Queensland, 4575, Australia
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Research Site 1
RECRUITINGMontreal, Quebec, H3A2B4, Canada
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Research Site 1
RECRUITINGUlm, 89081, Germany
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Research Site 1
RECRUITINGMilan, 20162, Italy
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Research Site 1
RECRUITINGLeiden, 2333, Netherlands
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Research Site 1
RECRUITINGAuckland, 1010, New Zealand
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Research Site 1
RECRUITINGValencia, 46026, Spain
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Research Site 2
RECRUITINGLiverpool, New South Wales, 2170, Australia
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Research Site 2
NOT_YET_RECRUITINGCalgary, Alberta, T2N4Z6, Canada
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Research Site 2
RECRUITINGMünchen, 80336, Germany
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Research Site 2
RECRUITINGRoma, 00168, Italy
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Research Site 2
RECRUITINGMadrid, 28034, Spain
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Research Site 3
RECRUITINGAuchenflower, Queensland, 4066, Australia
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Research Site 3
RECRUITINGEdmonton, Alberta, T6G2G4, Canada
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Research Site 3
RECRUITINGBarcelona, 08035, Spain
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Research Site 4
RECRUITINGMelbourne, Victoria, 3004, Australia