Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) (NCT03981575)
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
🩺 Plain-English Summary (8th-Grade Level)
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward. Funding Source- FDA OOPD
Who can join: Review the eligibility criteria below.
Location: University of California, San Diego — La Jolla, California
Age range: 18 Years to 70 Years
📋 "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 (17 Sites)
- University of California, San Diego — La Jolla, California
- University of California, Los Angeles — Los Angeles, California
- University of Colorado - Denver — Denver, Colorado
- University of Florida — Gainesville, Florida
- University of Iowa — Iowa City, Iowa
- Kansas University Medical Center — Kansas City, Kansas
- University of Rochester — Rochester, New York
- Ohio State University — Columbus, Ohio
- Houston Methodist Neurological Institute — Houston, Texas
- Virginia Commonwealth University — Richmond, Virginia
- Université de Sherbrooke — Québec,
- Friedrich Baur Institute, Ludwig-Maximilians-Universität München — München,
- Centro Clinico NeMO — Milan,
- Radboud University Medical Center — Nijmegen,
- University of Auckland — Auckland,
- St. George's, University of London — London,
- University College London — London,
📞 Contact the Study Coordinator
Phone: (804) 828-6318
Email: jennifer.raymond@vcuhealth.org
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
- Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy — Liverpool, New South Wales
- 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
- 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.