Roche Pharmaceuticals
The GENERATION HD2 study will evaluate the safety, biomarkers and efficacy of tominersen treatment given intrathecally once every 16-weeks over a period of 25 months (approx. two years). Participants will be randomized to one of three treatment study arms: 60mg, 100mg, or placebo. This means for every two participants randomized to tominersen, one will receive placebo. The study is designed to test the potential effects of tominersen compared to placebo. The study is “double-blind” meaning neither the participant nor his/her investigator or site staff will know which study arm the participant is assigned.
Status
Study Open – Recruiting
Formal Name
A Phase II, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington’s Disease.
Study Type
Interventional (Clinical Trial)
Purpose
This study will evaluate the efficacy, safety, and biomarker effects of tominersen compared with placebo in patients with prodromal and early manifest Huntington’s disease (HD).
Canadian Locations and Contact Information:
Alberta
Location: University of Alberta Hospital, Edmonton
Status: Recruiting
Quebec
Location: Location: Montréal Neurological Institute; Clinically Research Unit, Montréal
Status: Recruiting
Inclusion Criteria
Huntington 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 (eligibility that would exclude you from this study)
- 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, aspiring (unless </= 81 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, apixaban and heparin
- History of gene therapy, cell transplantation, or brain surgery
- 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
Source
For more detailed information on this study and others and for the most recent updates, please go to clinicaltrials.gov. ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.