Summary

for people ages 25-65 (full criteria)
at La Jolla, California and other locations
study started
estimated completion

Description

Summary

This study will evaluate the efficacy, safety, and biomarker effects of RO7234292 (RG6042) compared with placebo in patients with manifest Huntington's disease (HD).

Official Title

A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease

Keywords

Huntingtons Disease HD, RG6042, intrathecal, ASO, antisense oligonucleotide Huntington Disease

Eligibility

You can join if…

Open to people ages 25-65

  • Manifest HD diagnosis, defined as a DCL score of 4
  • Independence Scale (IS) score >= 70
  • Genetically confirmed disease by direct DNA testing with a CAP score >400
  • Clinical assessment to ensure individual has intact functional independence at baseline to maintain self-care and core activities of daily living (ADLs).

You CAN'T join if...

  • Any serious medical condition or clinically significant laboratory, or vital sign abnormality or claustrophobia at screening that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
  • Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug

Locations

  • University of California San Diego accepting new patients
    La Jolla California 92093 United States
  • SC3 Research Group, Inc accepting new patients
    Pasadena California 91105 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Hoffmann-La Roche
ID
NCT03761849
Phase
Phase 3
Study Type
Interventional
Last Updated