HDSA FAQ on the Roche/Genentech RG6042 program
HDSA's frequently asked questions factsheet about the huntingtin-lowering trial ASO program was so good we stole it (with permission)
By Leora Fox October 19, 2018 Edited by Dr Ed Wild Originally published on October 18, 2018
On Wednesday, September 26th, HDSA hosted a webinar presented by Genentech/Roche in which representatives from their Huntington’s disease team shared details and answered questions about upcoming Genentech/Roche clinical trials for HD. HDSA received hundreds of questions before, during, and after the webinar. Here HDSA’s staff scientists, with guidance from Genentech/Roche, have tried to address the most frequently-asked questions.
What Are The Two Upcoming Huntington’s Disease Clinical Trials Being Conducted By Genentech/Roche?
Can you tell me the basics about the planned GENERATION-HD1 study that will test huntingtin-lowering drug RG6042?
The planned GENERATION-HD1 will be a large Phase 3 study to test whether the Genentech/Roche huntingtin-lowering therapy RG6042 (formerly IONIS-Htt-Rx) is safe and effective in HD. It will involve monthly clinic visits for 25 months, with lumbar punctures (spinal tap) and other assessments each time.
660 people age 25-65, with manifest HD will be recruited. 220 will receive placebo every month, 220 will receive RG6042 every month, and 220 will receive RG6042 every other month with placebo on alternate months.
Upon study completion, the plan (if approved by health authorities) is to offer eligible participants in all groups the choice to receive RG6042 monthly or every other month (this is known as an Open-Label Extension Study).
What about the other study that isn’t testing a drug – the HD Natural History Study?
The HD Natural History Study does not involve a drug treatment. It will monitor people with early manifest HD by testing their symptoms and measuring their mutant huntingtin levels over time. It will last for 16 months and includes an initial screening, 4 clinic visits with lumbar punctures and other assessments (at baseline and at months 3, 9, and 15), and 2 phone check-ups (at months 6 and 12).
Around 100 people age 25-65 will be recruited.
Upon study completion, the plan (if approved by health authorities) is to offer eligible participants who complete the study the choice to begin receiving RG6042 (this is known as an Open-Label Extension Study).
Where And When Are These Studies Happening?
Where are the clinics? When do you expect to name them?
Genentech/Roche has devoted many months to identifying clinical sites. As of October 3rd, 2018, none have yet been confirmed, but the first announcements are likely to occur before the end of 2018.
When will the studies begin?
The first sites to be approved are expected to begin recruiting in late 2018 (for the HD Natural History Study) and early 2019 (for GENERATION-HD1).
The sites will not begin recruiting all at once, because there are different approval processes for each individual institution. However, HDSA will update the information on www.hdtrialfinder.org as it becomes available.
The Genentech Trial Information Support Line can also be contacted for more information (888) 662-6728 (Hours: Monday - Friday 5am - 5pm PT)
How many sites will be in the US?
There are no approved sites yet. For the HD Natural History Study, there will be up to 17 sites in the US, UK, Germany and Canada. For the GENERATION HD1 study there are expected to be up to 80-90 sites worldwide in approximately 15 countries, including the US.
Are all US sites HDSA Centers of Excellence?
No, but some are likely to be, because participating clinics need to be research centers capable of performing all the procedures involved in the trials. A variety of factors go into site selection, including assessments on experience with HD clinical studies, clinic infrastructure capacity to run the study and usual site activities, ability to operationalize the study as quickly and completely as possible, the patient population, and geographic location. HDSA does not have any control over site selection for these trials.
Can my clinic apply to be a site?
Genentech/Roche has informed HDSA that it is not looking for additional sites at this time, but your interest can be provided for future consideration. Contact the Genentech Trial Information Support Line at (888) 662-6728 (Hours: Monday - Friday 5am - 5pm PT).
Will patients be required to live within a certain distance of a site to be able to participate?
There is no specific distance requirement or catchment area for these trials, but the travel burden will likely be considered during the screening. A major move or a long-distance commitment could create additional stress on a participant and his/her loved ones. Excessive travel may also makes it more likely for someone to drop out of a trial, which could hamper the success of GENERATION-HD1 or the HD Natural History Study. Clinical studies are subject to international, national and local laws and regulations. Additionally, factors such as institutional site policies and health insurance may impact your ability to relocate and be accepted into one of the study sites. Eligibility and enrollment are ultimately decided by the study investigator at each site, who takes into account all these factors and may also wish to speak to you or your local HD specialist for more information.
To read the remainder of the article, go to: https://en.hdbuzz.net/265