Actively Recruiting

Age: 5Years +
All Genders
Healthy Volunteers
NCT05020002

Extracellular RNA Biomarkers of Myotonic Dystrophy

Led by Massachusetts General Hospital · Updated on 2025-11-24

215

Participants Needed

3

Research Sites

364 weeks

Total Duration

On this page

Sponsors

M

Massachusetts General Hospital

Lead Sponsor

B

Beth Israel Deaconess Medical Center

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Extracellular RNA Biomarkers of Myotonic Dystrophy

Who Can Participate

Age: 5Years +
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Subjects with DM1 or DM2 confirmed by genetic testing or clinical criteria
  • Able to provide informed consent or assent
  • Males and females aged 5 years and older for single biofluid collection
  • Males and females aged 14 years and older with DM1 for repeated measurements
  • Males and females aged 18 to 65 years for biofluid collection and muscle biopsy
Not Eligible

You will not qualify if you...

  • History of immunosuppression, coagulopathy, liver or kidney disease, HIV, or hepatitis B or C
  • Use of anti-platelet drugs within 7 days before blood draw or biopsy
  • Use of anticoagulants within 60 days before blood draw or biopsy
  • Active drug or alcohol use or dependence interfering with wound care
  • Women who are pregnant, planning pregnancy, or have a positive pregnancy test
  • Inability or unwillingness to provide written informed consent or assent

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 3 locations

1

Massachusetts General Hospital

Boston, Massachusetts, United States, 02129

Actively Recruiting

2

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States, 02215

Actively Recruiting

3

University of Texas Southwestern

Dallas, Texas, United States, 75390

Actively Recruiting

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Research Team

T

Tamkin Shahraki, MD

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

3

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Extracellular RNA Biomarkers of Myotonic Dystrophy | DecenTrialz