Biomarker Identification for Duchenne Muscular Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to find a less invasive way to track muscular dystrophies using urine and blood samples instead of muscle biopsies. Researchers seek to detect and measure the activity and severity of these conditions through these samples. Suitable candidates for this trial include males with Duchenne or Becker muscular dystrophy confirmed by genetic testing, who can provide consent and are not taking certain medications or have specific medical conditions.
As an unphased trial, this study offers participants the chance to contribute to groundbreaking research that could lead to less invasive diagnostic methods.
Do I have to stop taking my current medications for the trial?
The trial requires that you stop taking anti-platelet drugs 7 days before the blood draw and anticoagulants 60 days before the blood draw. Other medications are not specifically mentioned, so it's best to discuss with the trial team.
Why are researchers excited about this trial?
Researchers are excited about this trial because it explores the use of extracellular RNA as biomarkers for Duchenne Muscular Dystrophy (DMD). Unlike current treatments focusing on managing symptoms and slowing disease progression, identifying RNA biomarkers can provide a clearer, more precise way to monitor the disease and assess treatment effectiveness. This could potentially lead to earlier and more personalized intervention strategies, improving patient outcomes in ways that existing therapies cannot.
Who Is on the Research Team?
Thurman M. Wheeler, MD
Principal Investigator
Massachusetts General Hospital
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Sample Collection
Participants provide urine and blood samples for analysis of extracellular RNA biomarkers
Follow-up
Participants are monitored for safety and effectiveness after sample collection
What Are the Treatments Tested in This Trial?
Interventions
- None
Find a Clinic Near You
Who Is Running the Clinical Trial?
Massachusetts General Hospital
Lead Sponsor
Boston Children's Hospital
Collaborator
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