Purpose

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

Condition

Eligibility

Eligible Ages
Between 25 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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).

Exclusion Criteria

  • 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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
RO7234292 Q8W
RO4234292 is administered intrathecally every 8 weeks.
  • Drug: RO7234292
    Intrathecal injection
Experimental
RO7234292 Q16W
RO7234292 is administered intrathecally every 16 weeks. Participants in this arm will also receive placebo at alternate weeks to keep the blind.
  • Drug: RO7234292
    Intrathecal injection
  • Drug: Placebo
    Intrathecal injection
Placebo Comparator
Placebo
Placebo will be administered every 8 weeks by IT injection.
  • Drug: Placebo
    Intrathecal injection

Recruiting Locations

The University of Texas Health Science Center at Houston; McGovern Medical School
Houston, Texas 77030

More Details

NCT ID
NCT03761849
Status
Recruiting
Sponsor
Hoffmann-La Roche

Study Contact

Reference Study ID Number: BN40423 www.roche.com/about_roche/roche_worldwide.htm
888-662-6728 (U.S. and Canada)
global-roche-genentech-trials@gene.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.