Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy
Running, not enrolling
Conditions studied: Genetic Disorder
In brief
Background: \- Some kinds of muscular dystrophy affect the skeletal muscle membrane. In these conditions, the muscle membrane is more fragile. This affects how the muscles contract and relax, which causes movement problems. Researchers are looking at several muscle enzymes, or chemicals that affect how muscle cells function. By studying changes in these enzymes, they may be able to better understand how muscular dystrophy affects the cells. Researchers want to collect biomarkers (chemicals from blood samples) from people with fragile sarcolemmal muscular dystrophy. This information may provide better treatments for this condition. Objectives: \- To study biomarkers that may affect the muscles of people with fragile sarcolemmal muscular dystrophy. Eligibility: \- Individuals at least 18 years of age with fragile sarcolemmal muscular dystrophy. Design: * Participants will be screened with a medical history and physical exam. * Participants will be asked to come for four visits to the National Institutes of Health Clinical Center. The visits will be at least 2 months apart. Each visit will require participants to stay for 5 days at the clinical center. * During each visit, participants will provide frequent small blood samples. These samples will be collected while at rest and after physical exercise. * Participants will also have a physical therapy assessment. They will perform standard motor function tests and imaging tests (MRI, MRS). These tests may take up to 1 hour each time. * Treatment will not be provided as part of this study.
Key facts
- Study ID
- NCT01851447
- Run by
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- People needed
- 11
- Starts
- 2014-11-03
- Last updated by the study team
- 2026-07-30
Who can join
Age: 18 and older, up to 99. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Fail to meet the above inclusion criteria
- Are unable or unwilling to be examined
- Adults unable to provide their own consent
- Have active, on-going medical problems such as (e.g. diabetes, hypothyroidism, pancreatitis, anemia, cancer, renal, hepatic, pulmonary or cardiac disease) or who have undergone recent surgery (i.e. less than 8 days post-surgery)
- Pregnant females
- Currently taking any or a combination of anti-inflammatory drugs, statins or other drugs with known myotoxicity, narcotics
Where it is running
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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