Huntington's Disease clinical trials at UCSF

3 in progress, 2 open to eligible people

Showing trials for

  • Votoplam in Participants With Huntington's Disease

    open to eligible people ages 21-70

    The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.

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

    San Francisco, California and other locations

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

    Sorry, in progress, not accepting new patients

    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 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.

    San Francisco, California and other locations

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

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