Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
465 participants
OBSERVATIONAL
2015-02-20
2028-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Single biofluid collection
We will ask eligible volunteers to provide a single urine sample and undergo a single blood draw.
No interventions assigned to this group
Serial biofluid and muscle function testing
We will ask eligible volunteers to provide a urine sample, a blood sample, and undergo standard muscle function tests once every six months over a two-year period, and undergo pulmonary function tests and electrocardiogram once per year for two years.
No interventions assigned to this group
Biofluid and muscle tissue biopsy
We will ask eligible volunteers to provide a urine sample and undergo a muscle biopsy once.
No interventions assigned to this group
Ultrasound and myography testing
We will ask eligible volunteers to provide a single urine sample, a single blood draw, and undergo ultrasound and electrical impedance myography studies once.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Able to provide informed consent or assent for participation in the study.
* Demographic characteristics for single biofluid collection: Males and females age 5 years and older.
* Demographic characteristics for serial biofluid and muscle function testing: Males and females age 14 years and older with DM1.
* Demographic characteristics for biofluid and muscle biopsy: Males and females, ages 18-65 years.
Demographic characteristics for single biofluid collection, ultrasound, and myography: Males and females age 14 years and older.
Exclusion Criteria
* Medications and other drugs. 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 that, in the opinion of the biopsy surgeon, would interfere with post-procedure wound care.
* Other. Inability or unwillingness of the subject to give written informed consent.
5 Years
ALL
Yes
Sponsors
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Boston Children's Hospital
OTHER
Wake Forest University
OTHER
University of Pittsburgh
OTHER
Brigham and Women's Hospital
OTHER
Massachusetts General Hospital
OTHER
Responsible Party
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Thurman Wheeler, M.D
Physician Scientist
Principal Investigators
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Thurman M. Wheeler, MD
Role: PRINCIPAL_INVESTIGATOR
Massachusetts General Hospital
Locations
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Boston Children's Hospital
Boston, Massachusetts, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Wake Forest University
Winston-Salem, North Carolina, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Countries
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Central Contacts
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Facility Contacts
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References
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Antoury L, Hu N, Balaj L, Das S, Georghiou S, Darras B, Clark T, Breakefield XO, Wheeler TM. Analysis of extracellular mRNA in human urine reveals splice variant biomarkers of muscular dystrophies. Nat Commun. 2018 Sep 25;9(1):3906. doi: 10.1038/s41467-018-06206-0.
Antoury L, Hu N, Darras B, Wheeler TM. Urine mRNA to identify a novel pseudoexon causing dystrophinopathy. Ann Clin Transl Neurol. 2019 May 17;6(6):1106-1112. doi: 10.1002/acn3.777. eCollection 2019 Jun.
Other Identifiers
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2014P001727
Identifier Type: -
Identifier Source: org_study_id
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