Actively Recruiting
Biomarker Development for Muscular Dystrophies
Led by Massachusetts General Hospital · Updated on 2025-11-24
465
Participants Needed
5
Research Sites
692 weeks
Total Duration
On this page
Sponsors
M
Massachusetts General Hospital
Lead Sponsor
B
Boston Children's Hospital
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, 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.
CONDITIONS
Official Title
Biomarker Development for Muscular Dystrophies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subjects with DM1 or DM2 based on genetic testing and/or clinical criteria, or controls without muscular dystrophy by history
- Able to provide informed consent or assent for participation in the study
- Males and females age 5 years and older for single biofluid collection
- Males and females age 14 years and older for serial biofluid and muscle function testing with DM1
- Males and females age 18 to 65 years for biofluid collection and muscle biopsy
- Males and females age 14 years and older for single biofluid collection, ultrasound, and myography
You will not qualify if you...
- History of immunosuppression, coagulopathy, pre-existing liver or kidney disease, documented HIV, or hepatitis B/C
- 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 interfering with post-procedure care
- Inability or unwillingness to give written informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Active, Not Recruiting
2
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
3
Massachusetts General Hospital
Boston, Massachusetts, United States, 02129
Actively Recruiting
4
Wake Forest University
Winston-Salem, North Carolina, United States, 27157
Actively Recruiting
5
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
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
4
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