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Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

NCT03867435 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 4

Last updated 2021-11-04

No results posted yet for this study

Summary

Background:

Myotonic dystrophy is a long-term genetic disorder that affects muscle function. Symptoms include gradually worsening muscle loss and weakness. Muscles often contract and cannot relax. Researchers want to find out how various tests for DM1 or DM2 change over 2 years, to help them develop better tests for people with these diseases. Data and samples from this study will be shared with the Myotonic Dystrophy Clinical Research Network (DMCRN) investigators participating in the ongoing Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (ENDDM1) study

Objective:

To find better ways to assess how myotonic dystrophy type 1 or type 2 affects people.

Eligibility:

People ages 11 70 with DM1 or DM2

Design:

Participants will have 3 study visits over 2 years. Participants may be admitted to the clinic. Each visit may last up to a week and will include:

Medical history and physical exam

Blood, heart, and pregnancy tests

Questions about their disease

Breathing and muscle tests, including tests of movement, grip, and hand opening

Speech and swallowing exam

Magnetic resonance imaging (MRI). Participants will lie on a table that slides into a cylinder.

A magnetic field and radio waves will take pictures of the body. They may do a task during the scan. They may have a dye injected.

Pictures of chemicals in the brain or muscle taken in an MRI scanner

Thinking and memory tests

Sleep studies. Electrodes placed on the scalp will record the electrical activity of the brain.

Other devices on the body will measure heartbeat, breathing, movement, and oxygen.

Tests of electrical activity of muscles. Participants move their arms and legs with disks stuck on their skin.

Visits may also include:

Exam by a physician expert in stomach and bowel disorders

A piece of muscle and/or spinal fluid removed by needle

Sponsoring Institute: National Institute of Neurological Disorders and Stroke

...

Conditions

  • Myotonic Dystrophy Type-1
  • Myotonic Dystrophy Type-2

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Principal Investigators

  • Ami K Mankodi, M.D. · National Institute of Neurological Disorders and Stroke (NINDS)

Eligibility

Min Age
11 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-02-24
Primary Completion
2021-06-03
Completion
2021-11-02

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03867435 on ClinicalTrials.gov