F14 Collaborating with the community to conduct clinical trials in huntington’s disease: lessons from the tominersen phase iii generation HD1 study

BackgroundGENERATION HD1 (NCT03761849) is an ongoing Phase III study of tominersen, developed in collaboration with the Huntington’s disease (HD) community. In March 2021, dosing of tominersen in GENERATION HD1 was stopped, although participants continue to be assessed for safety and clinical outcomes.AimsTo describe the role of the HD community in successful site activation and recruitment in GENERATION HD1.MethodsSite start-up times and recruitment rates for GENERATION HD1 were compared with estimates from other trials conducted at the same sites and countries in the last 10 years.ResultsFeedback from the HD-COPE family coalition and global patient representatives were reflected in the study protocol design. The protocol was reviewed and formally endorsed by EHDN, HSG and EHA. Existing research networks and HD clinical expertise were leveraged, with input from Enroll-HD, HDSA, EHDN and HSG, to select study sites.Recruitment from January 2019 to April 2020 led to a total enrolment of 899 people with manifest HD. Average site activation times were faster in all countries than for other Roche studies conducted at the same sites in the last 10 years. The recruitment rate for GENERATION HD1 was nearly 2 times faster than the median rate for other non-Roche HD trials with >250 participants recruited, and was nearly 4 times faster than the median rate for other Roche trials in rare diseases conducted over the past 10 years.Additionally, collaboration with the HD community and knowledge sharing of mitigation measures were critical to mitigate the effects of the COVID-19 pandemic.ConclusionsCollaboration with the HD community resulted in successful and rapid activation and recruitment in GENERATION HD1.Roche would like to thank the patients and families who have participated and who are currently still participating in our research, and the ongoing partnership of the HD community.GENERATION HD1 is sponsored by F. Hoffmann-La Roche Ltd.