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Huntington's Disease clinical trials at UCSF

2 research studies open to eligible people

Huntington's disease is a condition that affects the brain and can cause problems with movement, thinking, and behavior. UCSF is recruiting for clinical trials that aim to test new treatments. One trial is testing the safety of a treatment called AMT-130 for people with early-stage Huntington's disease. Another trial is enrolling people in a global registry study to better understand this condition.

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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).

    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 20,000 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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