Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
NCT ID: NCT06131983
Last Updated: 2025-10-07
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
60 participants
INTERVENTIONAL
2024-02-26
2026-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ARO-DUX4
ARO-DUX4 for Injection
ARO-DUX4 for Injection
single or multiple doses of ARO-DUX4 by intravenous (IV) infusion
Placebo
(0.9%NaCl)
Placebo
calculated volume to match active treatment by IV infusion
Interventions
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ARO-DUX4 for Injection
single or multiple doses of ARO-DUX4 by intravenous (IV) infusion
Placebo
calculated volume to match active treatment by IV infusion
Eligibility Criteria
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Inclusion Criteria
* Clinical severity score between 3 and 8 (scale, 0 to 10)
* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader
* A 12-lead electrocardiogram (ECG) 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 12 weeks 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 12 weeks following the end of study or last dose of study medication, whichever is later.
Exclusion Criteria
* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) 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
16 Years
70 Years
ALL
No
Sponsors
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Arrowhead Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Research Site 2
Liverpool, New South Wales, Australia
Research Site 3
Auchenflower, Queensland, Australia
Research Site 1
Birtinya, Queensland, Australia
Research Site 4
Melbourne, Victoria, Australia
Research Site 2
Calgary, Alberta, Canada
Research Site 3
Edmonton, Alberta, Canada
Research Site 1
Montreal, Quebec, Canada
Research Site 2
München, , Germany
Research Site 1
Ulm, , Germany
Research Site 1
Milan, , Italy
Research Site 2
Roma, , Italy
Research Site 1
Leiden, , Netherlands
Research Site 1
Auckland, , New Zealand
Research Site 3
Barcelona, , Spain
Research Site 2
Madrid, , Spain
Research Site 1
Valencia, , Spain
Research Site
Bangkok, , Thailand
Countries
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Central Contacts
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Other Identifiers
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ARODUX4-1001
Identifier Type: -
Identifier Source: org_study_id
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