ClinicalTrials.gov ID: NCT05686551
Ages Eligible: 25 to 50 Years
Sexes Eligible: All
Study Phase: Phase 2
Study Completion (Estimated): 2027-04-01
Principal Investigator: Pinky Agarwal, MD, FAAN
Contact Information:
EvergreenHealth Research Department
425-899-5385
EvergreenResearch@evergreenhealthcare.org
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.
Eligibility Criteria:
Key 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
Key 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
