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Huntington's Disease clinical trials at UCSF

2 research studies open to eligible people

Huntington's disease is a brain disorder that affects a person's ability to think, talk, and move. UCSF is studying a global group of people with this disease in the Enroll-HD trial. We're also testing a new treatment called AMT-130 in adults with early signs of the disease.

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  • (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

    open to eligible people ages 25-65

    This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase I/II, randomized, multicenter, multiple dose, double-blind, imitation surgery, first-in-human (FIH) study. Cohort 3 participants will receive either high or low dose (1:1 randomization). Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab.

    San Francisco, California and other locations

  • Enroll -HD: A Prospective Registry Study in a Global Huntington's Disease Cohort

    open to eligible people ages 18 years and up

    Enroll-HD is a longitudinal, observational, multinational study that integrates two former Huntington's disease (HD) registries-REGISTRY in Europe, and COHORT in North America and Australasia-while also expanding to include sites in Latin America. More than 30,000 participants have now enrolled into the study. With annual assessments and no end date, Enroll-HD has built a large and rich database of longitudinal clinical data and biospecimens that form the basis for studies developing tools and biomarkers for progression and prognosis, identifying clinically-relevant phenotypic characteristics, and establishing clearly defined endpoints for interventional studies. Periodic cuts of the database are now available to any interested researcher to use in their research - visit to learn more.

    San Francisco, California and other locations

Our lead scientists for Huntington's Disease research studies include .

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