A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Patients With Manifest Huntington's Disease
- Conditions
- Huntington's Disease
- Registration Number
- JPRN-jRCT2080224757
- Lead Sponsor
- Chugai Pharmaceutical Co., Ltd.
- Brief Summary
The study treatment was terminated early at the discretion of the sponsor based on an independent review committee (iDMC) recommendation, and the primary efficacy endpoint of the study was not met. The results of secondary efficacy endpoints were in general consistent with the primary analysis.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 791
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).
- Any serious medical condition or clinically significant laboratory, or vital sign abnormality 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
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method efficacy<br>- Change from Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) [ Time Frame: Baselined, Week 101 ].<br>- Change From Baseline in the TFC Score [ Time Frame: Baseline, Week 101 ]
- Secondary Outcome Measures
Name Time Method