Status:
ACTIVE_NOT_RECRUITING
Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy
Lead Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborating Sponsors:
National Institute of Neurological Disorders and Stroke (NINDS)
National Institutes of Health Clinical Center (CC)
Conditions:
Genetic Disorder
Eligibility:
All Genders
18-99 years
Brief Summary
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 c...
Detailed Description
Objectives: the aim of this protocol is to identify biomarker and clinical correlates of changes in the barrier function of skeletal muscle membrane (i.e. cell membrane permeability) before and after ...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Aged 18 or older
- Have a confirmed genetic diagnosis of one of the FSMDs or have a clinical phenotype consistent with one of the FSMDs
- Be able to travel to the NIH Clinical Center at the NIH for studies
- Able to commit to multiple 5 day stays at the NIH Clinical Center
- Established primary care physician
- Ambulant: able to walk 10 meters or 33 feet without walking aids or orthotics
- EXCLUSION CRITERIA:
- 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
Exclusion
Key Trial Info
Start Date :
November 3 2014
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT01851447
Start Date
November 3 2014
Last Update
December 31 2025
Active Locations (1)
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1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892