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“Clinical Trial Ready” Studies

In CADASIL, a “clinical trial ready” study typically refers to a natural history, registry, or biomarker study designed to help prepare researchers for future treatment trials. Because CADASIL is a rare disease that progresses slowly and varies significantly between patients, researchers first need a clearer understanding of how the disease evolves over time and which measurements are most reliable for tracking progression. These studies help identify and follow genetically confirmed CADASIL patients, collect MRI and biomarker data, standardize cognitive and neurological assessments, and develop biobanks of blood, DNA, and imaging samples. The ultimate goal is to determine which patients may be best suited for future clinical trials, what outcomes should be measured, and how researchers can most effectively evaluate whether a therapy is working.

Several ongoing studies in the United States are helping build this “clinical trial ready” foundation for CADASIL research. At present, several studies are listed as “recruiting” on clinicaltrials.gov. Contact information for each study is available below, and interested individuals are encouraged to reach out directly to the participating centers for specific details regarding eligibility and participation. Together, these efforts are helping researchers prepare for future therapies aimed at targeting NOTCH3 dysfunction, protein aggregation, vascular smooth muscle injury, and other underlying disease mechanisms.