Status:

RECRUITING

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy

Lead Sponsor:

Massachusetts General Hospital

Conditions:

Myotonic Dystrophy Type 1

Eligibility:

All Genders

18+ years

Brief Summary

Myotonic dystrophy is associated with central sleep apnea, excessive daytime sleepiness, diminished working memory, impaired visuospatial skills, and deficits in problem-solving skills. Cerebrospinal...

Eligibility Criteria

Inclusion

  • Subjects with DM1 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).
  • Unaffected subjects are unknown to have myotonic dystropphy or any other muscular dystrophy by history and may have had no genetic testing.
  • Clinical indicators of current status, as measured within 30 days of study start: Able to provide informed consent or assent for participation in the study.
  • Demographic characteristics (e.g., biologic sex, age): Males and females age 18 years and older.

Exclusion

  • Medical history of any of the following. State of immunosuppression; pre-existing liver or kidney disease; documented HIV positive; documented hepatitis B and/or C positive.
  • Medications and other drugs. Use of anticoagulants within 60 days prior to lumbar puncture and/or blood draw. Use of anti-platelet drugs within 7 days prior to blood draw.
  • Contraindications to MRI. The presence of any metal within the body, which would include any medical device containing metal, such as a pacemaker, defibrillator, some heart valves or stents, artificial joint, aneurysm clip, or inner ear device, a history of working with sheet metal, or an injury with metal shrapnel; pregnancy, due to effects of MRI on unborn children.
  • Contraindications to Lumbar Puncture. Evidence of increased intracranial pressure or active infection on exam; platelets less than 50,000.
  • Other. Inability or unwillingness of the subject to give written informed consent.

Key Trial Info

Start Date :

August 1 2022

Trial Type :

OBSERVATIONAL

Allocation :

ESTIMATED

End Date :

August 1 2028

Estimated Enrollment :

88 Patients enrolled

Trial Details

Trial ID

NCT06075693

Start Date

August 1 2022

End Date

August 1 2028

Last Update

November 24 2025

Active Locations (1)

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Page 1 of 1 (1 locations)

1

Massachusetts General Hospital

Boston, Massachusetts, United States, 02129

Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy | DecenTrialz