Biomarker Development for Muscular Dystrophies (NCT05019625)
🩺 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, and some muscles in the arms and legs using tests called ultrasound and electrical impedance myography; both tests are painless and non-invasive. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human muscle diseases.
Who can join: Review the eligibility criteria below.
Location: Boston Children's Hospital — Boston, Massachusetts
Age range: 5 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 (5 Sites)
- Boston Children's Hospital — Boston, Massachusetts
- Brigham and Women's Hospital — Boston, Massachusetts
- Massachusetts General Hospital — Boston, Massachusetts
- Wake Forest University — Winston-Salem, North Carolina
- University of Pittsburgh — Pittsburgh, Pennsylvania
📞 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
- Extracellular RNA Biomarkers of Myotonic Dystrophy — 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.