GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease.
Purpose
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease
Condition
- Huntington Disease
Eligibility
- Eligible Ages
- Between 25 Years and 50 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Huntington's disease (HD) gene expansion mutation carrier status with a CAP score of 400-500 inclusive Either: - Prodromal HD (defined as DCL 2 to 3, Independence Scale (IS) ⩾70, and ⩾TFC8); or - Early manifest HD (defined as DCL 4, Independence Scale (IS) ⩾70, and ⩾TFC8); - Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2 - Study Companion
Exclusion Criteria
- Current or previous use of an ASO (including small interfering RNA) or any HTT lowering therapy (including tominersen) - Anti-platelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study, including, but not limited to, aspirin (unless </= 81 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban, and heparin - History of gene therapy, cell transplantation, or brain surgery - Hydrocephalus - Pregnancy or breastfeeding, or intention of becoming pregnant during the study or within 5 months after the final dose of study drug - 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
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Tominersen 60 mg |
|
|
Placebo Comparator Placebo |
|
|
Experimental Tominersen 100 mg |
|
Recruiting Locations
The University of Texas Health Science Center at Houston; McGovern Medical School
Houston, Texas 77030
Houston, Texas 77030
More Details
- Status
- Recruiting
- Sponsor
- Hoffmann-La Roche
Study Contact
Reference Study ID Number: BN42489. https://forpatients.roche.com/888-662-6728 (U.S. Only)
global-roche-genentech-trials@gene.com