Huntington's Disease
Conditions
Interventions
investigational material(s)
Generic name etc : Tominersen (RO7234292)
INN of investigational material : tominersen
Therapeutic category code : 119 Other agents affecting central nervous system
Dosage
Sponsors
Chugai Pharmaceutical Co., Ltd.
F Hoffman-La Roche
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy - Change from Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) [ Time Frame: Baselined, Week 101 ]. - Change From Baseline in the TFC Score [ Time Frame: Baseline, Week 101 ] | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy - Change From Baseline in Total Motor Score (TMS) [ Time Frame: Baseline, Week 101 ] - Change From Baseline in Symbol Digit Modalities Test (SDMT) [ Time Frame: Baseline, Week 101 ] - Change From Baseline in Stroop Word Reading (SWR) Test [ Time Frame: Baseline, Week 101 ] - Change From Baseline in the Clinical Global Impression, Severity Scale (CGI-S) [ Time Frame: Baseline, Week 101 ] - Percentage of Patients with a Decrease From Baseline of >=1 point on the TFC [ Time Frame: Baseline, Week101 ] - Percentage of Patients With a Decline From Baseline of >=1.2 Points on the cUHDRS [ Time Frame: Baseline, Week 101 ] - Percentage of Patients With an Unchanged or Improved Score on the Clinical Global Impression, Change Scale (CGI-C) Score [ Time Frame: Baseline to Week 101 ] safety - Percentage of Participants with Adverse Events [ Time Frame: Up to 117 Weeks (29 months) ] - Change From Baseline in Montreal Cognitive Assessment (MoCA) [ Time Frame: Screening, Baseline, Weeks 5, 21, 37, 53, 69, 85 and 101 and early treatment termination ] - Percentage of Participants With Suicidal Ideation or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) Score [ Time Frame: Screening, Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, end of treatment for early treatment termination and at early treatment termination ] pharmacokinetics - Concentration of RO7234292 in Plasma [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination ] - Trough Concentration of RO7234292 in CSF [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination ] other - Incidence of Anti-Drug Antibodies (ADAs) [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, and 101 and early treatment termination and safety follow-up visit ] - Titer and Antibody Subtype, determined if ADAs are Identified [ Time Frame: Baseline, Weeks 13, 21, 37, 53, 69, 85, | — |
Countries
Asia except Japan, Europe, Japan, North America, Oceania, South America
Outcome results
None listed