Huntington's Disease clinical trials at UCSF

• 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 www.enroll-hd.org/for-researchers/access-data/ to learn more.

  San Francisco, California and other locations

• Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington 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, dose escalation, double-blind, imitation surgery, first-in-human (FIH) study.

  San Francisco, California and other locations

Last updated: January 8, 2021