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Clinical Phenotype of CADASIL 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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Completed
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NCT ID: NCT02821780 Completed - Clinical trials for Cardiovascular Disease

CADASIL Disease Discovery

Start date: October 18, 2016
Phase:
Study type: Observational

Cerebral autosomal dominant arteriopathy with subcortical infarct (CADASIL) is a lethal disease caused by a gene mutation that affects arteries in the brain. Symptoms include migraines, strokes, memory loss, and dementia. There are no treatments. Researchers want to study people who have CADASIL to learn more about it. Objectives: To learn more about CADASIL by studying people who have it. Eligibility: People ages 18-100 who were diagnosed with CADASIL in the past 5 years and can make their own decisions Design: Participants will be screened in another NIH protocol. Participants will have 3 visits over 2 years. These may include: - Physical exam - Thinking and concentration tests - Blood tests - Skin biopsy: A small skin punch is removed from the arm or leg - Eye exam and eye imaging tests - Fluorescein angiogram: A catheter is placed in an arm vein. Dye is given through the catheter and travels to the eyes. - EndoPAT: A small clamp on the fingertip measures blood volume. - Cardio-ankle vascular index (CAVI): Artery stiffness is tested with blood pressure cuffs on the arms and legs. Soft electrodes on the skin measure heart signals. - Brain MRI or MRA: They lie on a table that slides in and out of a tube that takes pictures. They may get a contrast agent in their vein. It brightens the brain so researchers can see where blood flows. - CT scan of the heart: They lie on a table that slides in and out of a machine that takes pictures. - They get contrast dye injected through a catheter. They may get a medicine that makes their blood vessels bigger or slows their heart rate.