TerminatedNot Applicable

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

Stopped: PI left the NIH

United States4 participantsStarted 2020-02-24

Plain-language 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 ...

Who can participate

Age range

11 Years – 70 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* INCLUSION CRITERIA: * Age 11 to 70 inclusive * Competent to provide informed consent and assent (consent of a parent or guardian will be required for pediatric participants) * Positive genetic test for DM1 or DM2 (Genetic testing for DM1 or DM2 may be determined after enrollment EXCLUSION CRITERIA: * Concurrent enrollment in a clinical trial or participation in an investigative drug trial within 6 months of study entry. * Concurrent pregnancy or planned pregnancy during the course of the study. * Concurrent medical condition that would, in the opinion of the investigator or clinical evaluator, compromise performance on study measures: * Clinically significant infections or medical illness within 30 days from study entry. * History of, or abnormal laboratory values indicative of, significant medical, neurologic (other than DM1 or DM2), or psychiatric disorders. * A recent history (30 days prior to study entry) of any of the following conditions on routine blood screening: white blood cells \< 3000, platelets \< 100,000, hematocrit \< 30%, symptomatic liver disease with serum albumin \< 3 g/L, or creatinine \> 1.5 mg%. * Any of the following medical conditions: uncontrolled or insulin-dependent diabetes mellitus, congestive heart failure, symptomatic cardiomyopathy, symptomatic coronary artery disease, cancer (other than skin cancer) within 5 years from study entry, multiple sclerosis, or other serious medical illness. * Other diseases that mimic the signs or symptoms of DM…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

to characterize baseline status and disease progression over two years in the handgrip strength by dynamometry of DM1 and DM2 patients.

Timeframe: one-year and two-years

Trial details

NCT IDNCT03867435

SponsorNational Institute of Neurological Disorders and Stroke (NINDS)

Sponsor typeNIH

Study typeOBSERVATIONAL

Primary completion2021-06-03

View on ClinicalTrials.gov More Myotonic Dystrophy Type-1 trials