NIH-funded research R01 grant

Extracellular vesicle biomarkers to monitor DM1 using urine and blood samples

Official title: Prospective natural history study of extracellular vesicle metabolimic biomarkers of myotonic dystrophy

Funded at Massachusetts General Hospital · NIH-11494099 · Data updated 2026-11-12

A study looking for easy, noninvasive biomarkers in urine and blood to track disease activity in people with myotonic dystrophy type 1.

More Myotonic Dystrophy research and trials

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionMassachusetts General Hospital
NIH-funded
Lab location1 site (Boston, United States)
Project startSeptember 1, 2026
Project endAugust 31, 2030
Funding year2026
Award (this year)$400,000
NIH project number1R01FD008236-01A1
Project IDNIH-11494099 on NIH RePORTER

Who could benefit from this research

Could help

Patients with myotonic dystrophy type 1 who are able to participate in regular follow-up visits.

Less likely to help

Patients who cannot participate in serial sample collection or have contraindications to blood/urine collection may not benefit.

What this research studies

This project follows people with DM1 over time to see if molecules in extracellular vesicles found in urine and blood reflect how the disease is progressing or responding to treatment. Researchers will collect samples at multiple visits and analyze vesicle-related RNAs, proteins, and metabolites to identify reliable biomarkers. The goal is to enable frequent, painless monitoring that could reduce the need for muscle biopsies in future trials. Participation would involve scheduled visits, sample collection, and simple questions about health and symptoms. If successful, this approach could simplify tracking disease status and drug effects for patients and clinicians.

Why it matters

Potential benefit: If successful, patients could benefit from less invasive monitoring and faster assessment of treatment effects.

How similar studies have gone: Similar biomarker approaches have shown promise in other diseases, but this DM1-specific urinary EV metabolomic biomarker work is still being tested for its effectiveness.

Where this research is happening

  • Massachusetts General Hospital
    Boston, United States
    Active

Researchers

  • Principal investigator: Wheeler, Thurman M — Massachusetts General Hospital
  • Study coordinator: Wheeler, Thurman M

Related research

Other NIH-funded projects on the same topics.

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.