Extracellular RNA Biomarkers of Myotonic Dystrophy (NCT05020002)
🩺 Plain-English Summary (8th-Grade Level)
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (5 items) and avoid the listed exclusions (5 items).
Location: Massachusetts General Hospital — Boston, Massachusetts
Age range: 5 Years
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Medical history of any of the following. State of immunosuppression; coagulopathy; pre-existing liver or kidney disease; documented HIV positive; documented hepatitis B and/or C positive.
- Medications and other drugs. Use of anti-platelet drugs within 7 days prior to blood draw or biopsy; use of anticoagulants within 60 days prior to blood draw or biopsy; active drug or alcohol use or dependence that, in the opinion of the biopsy surgeon, would interfere with post-procedure wound care.
- Other. Women that are pregnant, or intend to become pregnant, prior to the biopsy; urine pregnancy test that is positive; inability or unwillingness of the subject to give written informed consent.
- Other. Inability or unwillingness of the subject to give written informed consent or assent.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (3 Sites)
- Massachusetts General Hospital — Boston, Massachusetts
- Beth Israel Deaconess Medical Center — Boston, Massachusetts
- University of Texas Southwestern — Dallas, Texas
📞 Contact the Study Coordinator
Phone: (617) 726-7506
Email: tshahraki@mgh.harvard.edu
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
- Biomarker Development for Muscular Dystrophies — Boston, Massachusetts
- Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry — Rochester, New York
- Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy — Winston-Salem, North Carolina
- Myotonic Dystrophy Family Registry — Oakland, California
- A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 — Gainesville, Florida
Explore by condition: All Myotonic Dystrophy 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.