Actively Recruiting
Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Led by Arrowhead Pharmaceuticals · Updated on 2026-02-06
60
Participants Needed
17
Research Sites
144 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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). In Part 1 of the study, participants will receive one dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.
CONDITIONS
Official Title
Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed facioscapulohumeral muscular dystrophy Type 1 (FSHD1)
- Clinical severity score between 3 and 8 on a scale of 0 to 10
- Eligible lower extremity muscle for biopsy confirmed by MRI
- Normal 12-lead electrocardiogram (ECG) at screening
- Use of highly effective contraception during the study and for at least 12 weeks after last dose for participants of childbearing potential and their partners
- Males must not donate sperm during the study and for at least 12 weeks after last dose
You will not qualify if you...
- Positive test for HIV infection at screening
- Positive test for hepatitis B (HBV) or hepatitis C (HCV) at screening
- Uncontrolled hypertension
- Severe cardiovascular disease
- History of thrombotic events
- Platelet count below normal limits at screening
- History or presence of hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease
- Inability to walk
- Use of hormone-based contraceptives
- Any contraindication to muscle biopsy or MRI
- Additional criteria may apply per protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 17 locations
1
Research Site 2
Liverpool, New South Wales, Australia, 2170
Actively Recruiting
2
Research Site 3
Auchenflower, Queensland, Australia, 4066
Actively Recruiting
3
Research Site 1
Birtinya, Queensland, Australia, 4575
Actively Recruiting
4
Research Site 4
Melbourne, Victoria, Australia, 3004
Actively Recruiting
5
Research Site 2
Calgary, Alberta, Canada, T2N4Z6
Not Yet Recruiting
6
Research Site 3
Edmonton, Alberta, Canada, T6G2G4
Actively Recruiting
7
Research Site 1
Montreal, Quebec, Canada, H3A2B4
Actively Recruiting
8
Research Site 2
München, Germany, 80336
Actively Recruiting
9
Research Site 1
Ulm, Germany, 89081
Actively Recruiting
10
Research Site 1
Milan, Italy, 20162
Actively Recruiting
11
Research Site 2
Roma, Italy, 00168
Actively Recruiting
12
Research Site 1
Leiden, Netherlands, 2333
Actively Recruiting
13
Research Site 1
Auckland, New Zealand, 1010
Actively Recruiting
14
Research Site 3
Barcelona, Spain, 08035
Actively Recruiting
15
Research Site 2
Madrid, Spain, 28034
Actively Recruiting
16
Research Site 1
Valencia, Spain, 46026
Actively Recruiting
17
Research Site
Bangkok, Thailand, 10700
Withdrawn
Research Team
M
Medical Monitor
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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