Extracellular RNA Biomarkers of Myotonic Dystrophy

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Massachusetts General Hospital
Study ID
NCT05020002
Status
Recruiting
Apply to this trial

Conditions

  • Myotonic Dystrophy

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.

Key Dates

Start date
Dec 8, 2020
Status verified
Nov 2025
Primary completion
Dec 31, 2026
Completion
Dec 31, 2027

Study Design

Enrollment
215 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.

Primary Outcome Measure

Extracellular RNA splice variants in biofluids [ Time Frame: 4 years ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Beth Israel Deaconess Medical CenterBostonMassachusetts02215
Hilda Gutierrez, MD
[email protected]
Seward B. Rutkove, MD (PRINCIPAL_INVESTIGATOR)
Courtney E. McIlduff, MD (SUB_INVESTIGATOR)
Massachusetts General HospitalBostonMassachusetts02129
Tamkin Shahraki, MD
[email protected]
617-726-7506
Thurman M. Wheeler, MD (PRINCIPAL_INVESTIGATOR)
University of Texas SouthwesternDallasTexas75390
Karla Castro
[email protected]
214-456-3696
Tammy Ramm
[email protected]
214-867-5631
Susan T. Iannaccone, MD (PRINCIPAL_INVESTIGATOR)

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