Status:
RECRUITING
Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Lead Sponsor:
Arrowhead Pharmaceuticals
Conditions:
Facio-Scapulo-Humeral Dystrophy
Eligibility:
All Genders
16-70 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1...
Eligibility Criteria
Inclusion
- 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 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
- Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening
- 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
- Note: additional inclusion/exclusion criteria may apply per protocol
Key Trial Info
Start Date :
February 26 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2026
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT06131983
Start Date
February 26 2024
End Date
December 1 2026
Last Update
October 7 2025
Active Locations (17)
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1
Research Site 2
Liverpool, New South Wales, Australia, 2170
2
Research Site 3
Auchenflower, Queensland, Australia, 4066
3
Research Site 1
Birtinya, Queensland, Australia, 4575
4
Research Site 4
Melbourne, Victoria, Australia, 3004