Clareo Health | 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
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
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Hoffmann-La Roche
Age
25–50
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
DB Period:
HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive
Either:
* Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ≥70, and TFC ≥8); Or
* Early manifest HD (defined as DCL 4, IS ≥70, and TFC ≥8)
Total body weight \> 40 kilograms (kg) and a body mass index (BMI) within the range of 18-32 kilograms per meter square (kg/m\^2)
Study companion
OLE Period:
Participants must have completed the DB treatment period
Participants must remain in the DB Safety follow-up period until OLE period starts
You may not be if
DB Period:
Current or previous use of an antisense oligonucleotide (ASO) (including small interfering ribonucleic acid \[RNA\]) 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 milligrams per day \[mg/day\]), 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
OLE Period:
Early discontinuation from the DB treatment and the safety follow-up (SFU) periods
Pregnant or breastfeeding, or with the intention of becoming pregnant during the study or within the timeframe in which contraception is required
Current or previous use of an ASO other than tominersen (including small interfering RNA) or any other HTT-lowering therapy
Hydrocephalus
Received any active investigational treatment other than tominersen during or since completion of the DB treatment period
