NCT05020002 Extracellular RNA Biomarkers of Myotonic Dystrophy
| NCT ID | NCT05020002 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Massachusetts General Hospital |
| Condition | Myotonic Dystrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 215 participants |
| Start Date | 2020-12-08 |
| Primary Completion | 2026-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 215 participants in total. It began in 2020-12-08 with a primary completion date of 2026-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * 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. * Able to provide informed consent or assent for participation in the study. * Demographic characteristics for single biofluid collection: Males and females age 5 years and older (DM1, DM2, and non-DM). * Demographic characteristics for repeated measurements: Males and females age 14 years and older with DM1. * Demographic characteristics for biofluid and muscle biopsy: Males and females, ages 18-65 years. 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. * 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. * Other. Women that are pregnant, or intend to become pregnant, prior to the biopsy; urine pregnancy test that is positive; inability or unwillingness of the subject to give written informed consent. * Other. Inability or unwillingness of the subject to give written informed consent or assent.
Contact & Investigator
Thurman M. Wheeler, MD
PRINCIPAL INVESTIGATOR
Massachusetts General Hospital
Frequently Asked Questions
Who can join the NCT05020002 clinical trial?
This trial is open to participants of all sexes, aged 5 Years or older, studying Myotonic Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05020002 currently recruiting?
Yes, NCT05020002 is actively recruiting participants. Contact the research team at tshahraki@mgh.harvard.edu for enrollment information.
Where is the NCT05020002 trial being conducted?
This trial is being conducted at Boston, United States, Boston, United States, Dallas, United States.
Who is sponsoring the NCT05020002 clinical trial?
NCT05020002 is sponsored by Massachusetts General Hospital. The principal investigator is Thurman M. Wheeler, MD at Massachusetts General Hospital. The trial plans to enroll 215 participants.