A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease

Purpose

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

Condition

  • Huntingtons Disease

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
    Other names:
    • Tominersen
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
    Other names:
    • Tominersen
  • Drug: Placebo
    Intrathecal injection
Placebo Comparator
Placebo 		Placebo will be administered every 8 weeks by IT injection.
  • Drug: Placebo
    Intrathecal injection

More Details

Status
Completed
Sponsor
Hoffmann-La Roche

Study Contact