Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
ConditionDuchenne Muscular Dystrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age5+
SponsorMassachusetts General Hospital
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial population
Males ages 5 years and older with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD). In addition, male and female family members without known muscular dystrophy ages 18 and older also are invited to participate.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
100 Participants
are grouped into 1 trial groupGroup A: Biofluid collection
Trial outcomes
Primary outcomes
1
Extracellular RNA in biofluids
The extracellular RNA biomarkers in the muscular dystrophy groups will be evaluated and compared with the extracellular RNA content in control groups. Statistical analysis will be used to evaluate the sensitivity and specificity of these markers as measurements of disease activity and severity.
Time frame
4 yearsSponsors and contacts
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Massachusetts General Hospital
Lead sponsor
Boston Children's Hospital
Collaborator
Source ClinicalTrials.gov