Evaluating ARO-DUX4 for treating Facioscapulohumeral Muscular Dystrophy Type 1
A Phase1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
This study is testing a new treatment called ARO-DUX4 to see if it can safely help adults with Facioscapulohumeral Muscular Dystrophy Type 1 feel better.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 16 Years to 70 Years |
| Sex | All |
| Sponsor | Arrowhead Pharmaceuticals Industry-sponsored |
| Locations | 17 sites (Liverpool, New South Wales and 16 other locations) |
| Trial ID | NCT06131983 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-DUX4 in adults diagnosed with Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1). Participants will first receive a single dose of ARO-DUX4 or a placebo, followed by a second phase where they may receive four doses. The study includes MRI-guided muscle biopsies to evaluate the drug's effects. Those who complete the initial phases may have the opportunity to continue in an open-label extension study or participate in future clinical trials.
Who should consider this trial
Good fit: Ideal candidates are adults with genetically confirmed FSHD1 and a clinical severity score between 3 and 8.
Not a fit: Patients with HIV, hepatitis B, or hepatitis C infections may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the management of FSHD1 and enhance the quality of life for affected patients.
How similar studies have performed: While this approach is novel, similar studies targeting genetic muscular dystrophies have shown promise in early phases.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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
Where this trial is running
Liverpool, New South Wales and 16 other locations
- Research Site 2 — Liverpool, New South Wales, Australia (Recruiting)
- Research Site 3 — Auchenflower, Queensland, Australia (Recruiting)
- Research Site 1 — Birtinya, Queensland, Australia (Recruiting)
- Research Site 4 — Melbourne, Victoria, Australia (Recruiting)
- Research Site 2 — Calgary, Alberta, Canada (Not_yet_recruiting)
- Research Site 3 — Edmonton, Alberta, Canada (Recruiting)
- Research Site 1 — Montreal, Quebec, Canada (Recruiting)
- Research Site 2 — München, Germany (Recruiting)
- Research Site 1 — Ulm, Germany (Recruiting)
- Research Site 1 — Milan, Italy (Recruiting)
- Research Site 2 — Roma, Italy (Recruiting)
- Research Site 1 — Leiden, Netherlands (Recruiting)
- Research Site 1 — Auckland, New Zealand (Recruiting)
- Research Site 3 — Barcelona, Spain (Recruiting)
- Research Site 2 — Madrid, Spain (Recruiting)
- Research Site 1 — Valencia, Spain (Recruiting)
- Research Site — Bangkok, Thailand (Withdrawn)
Study contacts
- Study coordinator: Medical Monitor
- Email: ARO_DUX4@arrowheadpharma.com
- Phone: 626-304-3400
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.