Study of SRP-1001 in Adult and Adolescent Participants With Facioscapulohumeral Muscular Dystrophy Type 1 (NCT06131983)
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 (SRP-1001) in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SRP-1001 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of SRP-1001 or placebo. In Part 2 of the study, participants will receive 4 doses of SRP-1001 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 magnetic imaging resonance (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.
Who can join: Review the eligibility criteria below.
Location: Liverpool Hospital — Liverpool, New South Wales
Age range: 16 Years to 70 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (16 Sites)
- Liverpool Hospital — Liverpool, New South Wales
- Wesley Research Institute — Auchenflower, Queensland
- Sunshine Coast University Hospital — Birtinya, Queensland
- Alfred Health — Melbourne, Victoria
- University of Calgary — Calgary, Alberta
- University of Alberta — Edmonton, Alberta
- McGill University — Montreal, Quebec
- Universitätsklinikum München — München,
- Neurologische Universitätsklinik Am Rku — Ulm,
- Centro Clinico Nemo Milann — Milan,
- Fondazione Policlinico Universitario A. Gemelli — Roma,
- Centre For Human Drug Research — Leiden,
- New Zealand Clinical Research — Auckland,
- Hospital Universitario Vall d'Hebron — Barcelona,
- Hospital Universitario Ramon y Cajal — Madrid,
- Hospital Universitari l Politecnic La Fe de Valencia — Valencia,
📞 Contact the Study Coordinator
Phone: 1-888-SAREPTA (1-888-727-3782)
Email: SareptAlly@sarepta.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Muscular Dystrophy, Facioscapulohumeral
- Clenbuterol to Target DUX4 in FSHD — Kansas City, Kansas
- Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds) — Palo Alto, California
Explore by condition: All Muscular Dystrophy, Facioscapulohumeral clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.