Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

Description

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.

Conditions

Duchenne Muscular Dystrophy

Study Overview

Study Details

Study overview

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.

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Boston

Massachusetts General Hospital, Boston, Massachusetts, United States, 02129

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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.

Ages Eligible for Study

5 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Massachusetts General Hospital,

Thurman M. Wheeler, MD, PRINCIPAL_INVESTIGATOR, Massachusetts General Hospital

Study Record Dates

2024-05