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

Roche

Status and phase: Completed; Phase 3

Conditions: Huntingtons Disease

Treatments: Drug: RO7234292; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03761849

BN40423

GENERATION HD1 (Other Identifier)

Details and patient eligibility

About

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

Enrollment: 899 patients

Sex: All

Ages: 25 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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899 participants in 3 patient groups, including a placebo group

RO7234292 Q8W

Experimental group

Description:

RO4234292 is administered intrathecally every 8 weeks.

Treatment:

Drug: RO7234292

RO7234292 Q16W

Experimental group

Description:

RO7234292 is administered intrathecally every 16 weeks. Participants in this arm will also receive placebo at alternate weeks to keep the blind.

Treatment:

Drug: RO7234292

Drug: Placebo

Placebo

Placebo Comparator group

Description:

Placebo will be administered every 8 weeks by IT injection.

Treatment:

Drug: Placebo