Status:
TERMINATED
Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Myotonic Dystrophy Type-1
Myotonic Dystrophy Type-2
Eligibility:
All Genders
11-70 years
Brief 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. Re...
Detailed Description
Objective: Myotonic dystrophy type 1 (DM1) and type 2 (DM2) are the most common inherited skeletal myopathies in adults. RNA toxicity is the core disease mechanism, good molecular targets have been id...
Eligibility Criteria
Inclusion
- 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 DM1 or DM2. Coexistence of other neuromuscular disease.
- Thyroid dysfunction that is untreated (if on thyroid hormone replacement therapy, need to have adequate and stable replacement over the previous 6 months from study entry).
- Second or third degree heart block, atrial flutter, atrial fibrillation, ventricular tachycardia, or is receiving medication for the treatment of cardiac arrhythmia.
- Liver or kidney disease requiring ongoing treatment.
- Have a seizure disorder.
- Drug or alcohol abuse within 3 months of study entry (DSM-V criteria will be used for the diagnosis and level of a substance use disorder:
- Treatment with supplemental anabolic hormones (including testosterone, human recombinant growth hormone, human recombinant insulin like growth factor-1, other anabolic drug mixtures) during the previous 12 months from study entry.
- Note: non-ambulatory participants are not excluded but are limited to \<15% of enrollment. Individuals using a cane or walker will not be examined for gait, balance and mobility.
Exclusion
Key Trial Info
Start Date :
February 24 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
November 2 2021
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT03867435
Start Date
February 24 2020
End Date
November 2 2021
Last Update
November 4 2021
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892