Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy (NCT06138743)
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 (SRP-1003) in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years
🩺 Plain-English Summary (8th-Grade Level)
This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Who can join: Review the eligibility criteria below.
Location: Liverpool Hospital — Liverpool, New South Wales
Age range: 18 Years to 65 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 (35 Sites)
Showing the first 25 of 35 sites.
- Liverpool Hospital — Liverpool, New South Wales
- Sunshine Coast University Hospital — Birtinya, Queensland
- Royal Brisbane And Women's Hospital — Herston, Queensland
- Alfred Health — Melbourne, Victoria
- Nucleus Network Brisbane Clinic — Herston,
- Mater Hospital Brisbane — South Brisbane,
- Universitair Ziekenhuis Gent — Ghent,
- UZ Leuven — Leuven,
- University Of Calgary — Calgary,
- University Of Alberta — Edmonton,
- McGill University — Montreal,
- University Hospital of Angers — Angers,
- Centre Hospitalier Universitaire De Bordeaux — Bordeaux,
- Centre Hospitalier Universitaire De Lille — Lille,
- CHU de Nice - Hôpital Pasteur 2 — Nice,
- Assistance Publique Hôpitaux De Paris — Paris,
- Klinikum der Universität München AöR — Munich,
- Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. — Ulm,
- Fondazione Serena Onlus - Centro Clinico Nemo Brescia — Gussago,
- Centro Clinico Nemo (Milan) — Milan,
- Azienda Ospedaliera Universitaria Pisana (AOUP) — Pisa,
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma,
- New Zealand Clinical Research Christchurch — Christchurch,
- Hospital Universitario Torrecárdenas — Almería,
- Hospital Universitari Vall D Hebron — Barcelona,
📞 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 Myotonic Dystrophy 1
- A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 — Calgary, Alberta
- Study of ATX-01 in Participants With DM1 — Los Angeles, California
- Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) — La Jolla, California
- An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) — Calgary, Alberta
Explore by condition: All Myotonic Dystrophy 1 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.