Over the past several years, Roche has become a common name in households of HD families across the globe, most commonly through their investigations regarding tominersen. Over the past year, they have been focusing on other research in addition to their GENERATION-HD2 study.
Clinical Trials
Generation HD2
- Who is overseeing this study? Roche
- What’s its current status? Active
- Where can people participate? Opened in North America, Europe, South America and Oceania.
- Who’s potentially eligible? People aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD.
- What are they investigating? This study evaluates the safety, biomarkers and efficacy trends of different doses of the investigational drug tominersen (given as an injection by lumbar puncture) in people aged 25 to 50 with prodromal (very early subtle signs of HD) or early manifest HD.
Phase I Study of SPK-1001
- Who is overseeing this study? Roche and Huntington Study Group (HSG)
- What’s its current status? Active and recruiting.
- Where can people participate? United States
- Who’s potentially eligible? Adults ages 25-65 who have genetically confirmed HD and mild clinical symptoms.
- What are they investigating? This trial is assessing the safety, tolerability and preliminary efficacy of SPK-10001 in patients suffering from Huntington's Disease. SPK-10001 is a gene therapy that delivers genetic instructions into the brain cells to reduce mutant huntingtin that causes brain atrophy and clinical symptoms affecting motor function, cognition, and behavior for individuals with HD. In this study, the researchers will slowly increase the dose for each small group of participants to find the best dose of a study treatment for participants to receive.
POINT HD
- Who is overseeing this study? Roche
- What’s its current status? Active and recruiting
- Where can people participate? New Zealand and Australia
- Who’s potentially eligible? Adults ages 25-65 who have genetically confirmed HD and mild clinical symptoms as well as who carry a specific genetic variation in the HD gene targeted by the drug being investigated
- What are they investigating? This Phase I study is testing the safety and tolerability of a drug called RG6496 at different doses in patients suffering from HD. RG6496 is being developed with the goal of lowering levels of the faulty huntingtin protein, while keeping healthy huntingtin protein. It works by targeting a specific genetic variation that some people carry in their HD gene.
Observational Study
Epidemiologic Study of SNPs
- Who is overseeing this study? Hoffmann-La Roche
- What’s its current status? Active and recruiting
- Where can people participate? US, Canada, Argentina, Europe, And Australia/NewZealand.
- Who’s potentially eligible? Adults ages 25-60 who are confirmed HD carriers
- What are they investigating? This study aims to understand the frequency of different SNPs among individuals who are HD gene carriers.
