Biomarker Development for Muscular Dystrophies

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Massachusetts General Hospital
Study ID
NCT05019625
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
5 Years - N/A
Healthy Volunteers
Accepted

Study Details

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.

Key Dates

Start date
Feb 20, 2015
Status verified
Nov 2025
Primary completion
Jun 30, 2027
Completion
Jun 30, 2028

Study Design

Enrollment
465 participants (estimated)

Arms

  • Arm: Single biofluid collection
    We will ask eligible volunteers to provide a single urine sample and undergo a single blood draw.
  • Arm: 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.
  • Arm: Biofluid and muscle tissue biopsy
    We will ask eligible volunteers to provide a urine sample and undergo a muscle biopsy once.
  • Arm: 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.

Primary Outcome Measure

Extracellular RNA in biofluids [ Time Frame: 6 years ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115-
Brigham and Women's HospitalBostonMassachusetts02115
Anthony Amato, MD (PRINCIPAL_INVESTIGATOR)
Massachusetts General HospitalBostonMassachusetts02129
Tamkin Shahraki, MD
[email protected]
617-726-7506
Thurman M. Wheeler, MD (PRINCIPAL_INVESTIGATOR)
Paloma Gonzalez-Perez, MD, PhD (SUB_INVESTIGATOR)
Wake Forest UniversityWinston-SalemNorth Carolina27157
Constance Linville
[email protected]
336-716-4568
Araya Puwanant, MD (PRINCIPAL_INVESTIGATOR)
University of PittsburghPittsburghPennsylvania15213
Kerry M. Oddis, RN, CCRC
[email protected]
412-692-4918
Paula R. Clemens, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Boston, MA

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Boston Children's Hospital· Boston, MABrigham and Women's Hospital· Boston, MAMassachusetts General Hospital· Boston, MAWake Forest University· Winston-Salem, NCUniversity of Pittsburgh· Pittsburgh, PA

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