โš ๏ธ Medical Notice: If you are experiencing a medical emergency, call 911 or go to the nearest ER. This directory is an educational resource and not an emergency medical service.
๐Ÿ›๏ธ Verified NIH Protocol โ€ข Sourced via U.S. National Library of Medicine (Last Synced: 2026-08-11)

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy

๐ŸŸข Recruiting Phase 1 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: Sarepta Therapeutics, Inc.

๐Ÿฉบ Protocol Summary

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.

Primary Research Facility: Liverpool Hospital (Liverpool, New South Wales)

๐Ÿ“‹ Basic Study Criteria & Logistics Checklist

0 of 3 Checked

Review these 3 basic logistical and protocol criteria extracted from the public NIH record. (This is a preliminary logistical checklist, not an official clinical eligibility determination).

๐Ÿ“ Primary Site Map

Site: Liverpool Hospital โ€” Liverpool, New South Wales

๐Ÿ“ All Participating Trial Locations (35 Sites)

Multi-Center Trial

This protocol is enrolling participants across multiple clinical centers:

1. Liverpool Hospital โ€” Liverpool, New South Wales
2. Sunshine Coast University Hospital โ€” Birtinya, Queensland
3. Royal Brisbane And Women's Hospital โ€” Herston, Queensland
4. Alfred Health โ€” Melbourne, Victoria
5. Nucleus Network Brisbane Clinic โ€” United States
6. Mater Hospital Brisbane โ€” United States
7. Universitair Ziekenhuis Gent โ€” United States
8. UZ Leuven โ€” United States
9. University Of Calgary โ€” United States
10. University Of Alberta โ€” United States
11. McGill University โ€” United States
12. University Hospital of Angers โ€” United States
13. Centre Hospitalier Universitaire De Bordeaux โ€” United States
14. Centre Hospitalier Universitaire De Lille โ€” United States
15. CHU de Nice - Hรดpital Pasteur 2 โ€” United States
16. Assistance Publique Hรดpitaux De Paris โ€” United States
17. Klinikum der Universitรคt Mรผnchen AรถR โ€” United States
18. Deutsches Zentrum fรผr Neurodegenerative Erkrankungen e.V. โ€” United States
19. Fondazione Serena Onlus - Centro Clinico Nemo Brescia โ€” United States
20. Centro Clinico Nemo (Milan) โ€” United States
21. Azienda Ospedaliera Universitaria Pisana (AOUP) โ€” United States
22. Fondazione Policlinico Universitario Agostino Gemelli IRCCS โ€” United States
23. New Zealand Clinical Research Christchurch โ€” United States
24. Hospital Universitario Torrecรกrdenas โ€” United States
25. Hospital Universitari Vall D Hebron โ€” United States

๐Ÿ“„ Print Doctor Discussion Sheet

Preview and print a 1-page summary with 5 tailored questions for your physician.

๐Ÿ’ฐ Clinical Trial Costs & Insurance Coverage

Coverage for clinical trial participation varies depending on your health insurance policy, state legislation, and whether specific procedures are investigational research or routine medical care. Under federal law (Affordable Care Act ยง 2709), health insurers generally cannot drop coverage or refuse to pay for routine patient care costs incurred in approved clinical trials.

๐Ÿ’ก Always ask the study coordinator for the trial's official Informed Consent Form, which outlines all sponsor-funded research items versus procedures billed to your insurance.

๐Ÿ›๏ธ Official Government Provenance

NIH ClinicalTrials.gov Protocol Record

NCT Identifier: NCT06138743 โ€ข Last Synced: 2026-08-11
View on ClinicalTrials.gov โ†—
๐Ÿ“ž Call Desk ๐Ÿ“‹ Check Criteria