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Genetic Disorder clinical trials

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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NCT ID: NCT01851447 Active, not recruiting - Genetic Disorder Clinical Trials

Skeletal Muscle Biomarkers in People With Fragile Sarcolemmal Muscular Dystrophy

Start date: November 3, 2014
Phase:
Study type: Observational

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.