Examining urine and blood samples for Duchenne Muscular Dystrophy biomarkers
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
This study is testing if urine and blood samples can help track how Duchenne Muscular Dystrophy affects boys aged 5 and older, instead of using more invasive muscle biopsies.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Ages | 5 Years and up |
| Sex | All |
| Sponsor | Massachusetts General Hospital Academic / other |
| Locations | 2 sites (Boston, Massachusetts and 1 other locations) |
| Trial ID | NCT05016908 on ClinicalTrials.gov |
What this trial studies
This observational study aims to identify less invasive methods for measuring the activity and severity of Duchenne Muscular Dystrophy (DMD) by analyzing urine and blood samples instead of relying on muscle biopsies. The researchers will collect biofluids from male participants aged 5 years and older diagnosed with DMD or Becker Muscular Dystrophy (BMD), as well as control subjects without muscular dystrophy. The goal is to explore the potential of extracellular RNA biomarkers as indicators of disease progression and treatment response.
Who should consider this trial
Good fit: Ideal candidates include males aged 5 years and older with a confirmed diagnosis of DMD or BMD, as well as males and females aged 18 and older without muscular dystrophy.
Not a fit: Patients with a history of immunosuppression, coagulopathy, liver or kidney disease, or those who have used certain medications prior to the study may not benefit.
Why it matters
Potential benefit: If successful, this study could provide a non-invasive method for monitoring DMD, improving patient comfort and compliance.
How similar studies have performed: Other studies exploring non-invasive biomarkers for muscular dystrophies have shown promise, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subjects with DMD or BMD based on genetic testing. Control subjects are unknown to have any other muscular dystrophy by history and may have had no genetic testing. * Able to provide informed consent or assent for participation in the study. * Demographic characteristics for biofluid collection: Males age 5 years and older with DMD or BMD; males and females ages 18 years and older without muscular dystrophy. 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. * Use of anti-platelet drugs within 7 days prior to blood draw; use of anticoagulants within 60 days prior to blood draw. * Inability or unwillingness of the subject to give written informed consent.
Where this trial is running
Boston, Massachusetts and 1 other locations
- Boston Children's Hospital — Boston, Massachusetts, United States (Active_not_recruiting)
- Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: Thurman M. Wheeler, MD — Massachusetts General Hospital
- Study coordinator: Tamkin Shahraki, MD
- Email: tshahraki@mgh.harvard.edu
- Phone: 617-726-7506
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.