RECRUITING

Biomarker Development for Muscular Dystrophies

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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, and some muscles in the arms and legs using tests called ultrasound and electrical impedance myography; both tests are painless and non-invasive. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human muscle diseases.

Official Title

Biomarker Development for Muscular Dystrophies

Quick Facts

Study Start:2015-02-20
Study Completion:2027-06
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05019625

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.

Ages Eligible for Study:5 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:Yes
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Subjects with DM1 or DM2 based on genetic testing and/or clinical criteria (some subjects who have positive genetic testing may be asymptomatic, while other subjects who show characteristic clinical features may have declined to have genetic testing done). Control non-DM subjects are unknown to have DM or any other muscular dystrophy by history and may have had no genetic testing.
  2. * Able to provide informed consent or assent for participation in the study.
  3. * Demographic characteristics for single biofluid collection: Males and females age 5 years and older.
  4. * Demographic characteristics for serial biofluid and muscle function testing: Males and females age 14 years and older with DM1.
  5. * Demographic characteristics for biofluid and muscle biopsy: Males and females, ages 18-65 years.
  1. * 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.
  2. * 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.
  3. * Other. Inability or unwillingness of the subject to give written informed consent.

Contacts and Locations

Study Contact

Tamkin Shahraki, MD
CONTACT
617-726-7506
tshahraki@mgh.harvard.edu

Principal Investigator

Thurman M. Wheeler, MD
PRINCIPAL_INVESTIGATOR
Massachusetts General Hospital

Study Locations (Sites)

Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115
United States
Massachusetts General Hospital
Boston, Massachusetts, 02129
United States
Wake Forest University
Winston-Salem, North Carolina, 27157
United States
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
United States

Collaborators and Investigators

Sponsor: Massachusetts General Hospital

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2015-02-20
Study Completion Date2027-06

Study Record Updates

Study Start Date2015-02-20
Study Completion Date2027-06

Terms related to this study

Additional Relevant MeSH Terms

  • Myotonic Dystrophy
  • Duchenne Muscular Dystrophy
  • Becker Muscular Dystrophy
  • Facioscapulohumeral Muscular Dystrophy