The companies Genentech and Roche are working together to carry out a program of studies involving patients with Huntington’s disease (the huntingtin-lowering trial ASO program). One is a natural history study that doesn’t involve treatment, and the other will look at the effects of an investigational drug. They are also planning to carry out an open-label extension study, which will give patients the chance to receive the investigational drug in the long-term.
A lot of people want to know more about these studies, what they will involve, and who is eligible. To answer some of the questions, Genentech and Roche took part in a Q&A webinar hosted by HDSA, and some of the most frequently asked questions are now answered in a factsheet about the trials that you can find here, at HD Buzz.
The Generation-HD1 Study
The Generation-HD1 study is a Phase 3 clinical trial that will look at the effects of an investigational drug called RG6042 (previously IONIS-Htt-Rx) in people with Huntington’s disease, to see if it’s safe and effective. According to the Huntington Society of Canada, this drug has previously been studied in a Phase 1/2a trial, which found that it was safe and that higher doses could reduce the huntingtin protein, implicated in Huntington’s disease, by 40 to 60%.
The upcoming study is expected to recruit around 660 people with manifest Huntington’s disease who are aged between 25-65. These participants will be divided into three groups: one given RG6042 every month, another given it every other month (with a placebo on alternate months), and a third given a placebo each month. The study is planned to last 25 months, and people taking part will need to visit a clinic for testing monthly.
The Natural History Study
This study won’t involve drug treatment and instead focuses on monitoring the mutant huntingtin levels of people diagnosed with early manifest Huntington’s disease over time. Researchers aim to recruit about one hundred participants aged between 25 and 65 and will follow them for sixteen months, involving a planned four clinic visits and two phone calls.
The Extension Study
People who take part in either of these studies may be eligible for an Open-Label Extension Study, which would give them the option to receive RG6042 either every month or every other month. However, this study is still awaiting approval from health authorities.
For more detailed information about these studies and the recruitment process, follow the link at the top of the page to the Q&A article.