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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05016908
Other study ID # 2019P001504
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date November 30, 2019
Est. completion date May 2024

Study information

Verified date October 2023
Source Massachusetts General Hospital
Contact Tamkin Shahraki, MD
Phone 617-726-7506
Email tshahraki@mgh.harvard.edu
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 100
Est. completion date May 2024
Est. primary completion date November 2023
Accepts healthy volunteers
Gender All
Age group 5 Years and older
Eligibility 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.

Study Design


Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States Massachusetts General Hospital Boston Massachusetts

Sponsors (2)

Lead Sponsor Collaborator
Massachusetts General Hospital Boston Children's Hospital

Country where clinical trial is conducted

United States, 

References & Publications (2)

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. — View Citation

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. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary 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. 4 years
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