A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease.

• Status: Recruiting
• Trial end date: 2027-01-04
• Target enrollment: 0
• Participant gender: All

Summary

This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hoffmann-La Roche

Criteria

Key Inclusion Criteria

-HD gene expansion mutation carrier status. CAP score of 400-500, inclusive, where CAP is calculated as follows: Age x (CAG repeat length -33.66)

-Prodromal HD, defined as DCL 2 to 3, Total Motor Score (TMS) >6, Independence Scale (IS) = 100 (broadly equivalent to HD-ISS Stage 2), or early manifest HD, defined as DCL 4, TMS > 6, 100 >IS>/= 70, and TFC >/=8 (broadly equivalent to HD-ISS Stage 3)

Key Exclusion Criteria

• History of attempted suicide or suicidal ideation with plan (i.e., active suicidal ideation) that required hospital visit and/or change in level of care within 12 months prior to screening

• Active psychosis, confusional state, or violent behavior, including aggression that could cause harm to self or others, over the 12 weeks prior to screening

• Any serious medical condition or clinically significant laboratory or vital sign abnormality or claustrophobia at screening that, in the investigator's judgment, precludes the individual's safe participation in and completion of the study

• Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug