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

Supporting Huntington’s Disease Families Through the Ups and Downs of Clinical Trials

“…For GENERATION HD1, this was very much the case. Almost 800 people with early manifest HD were enrolled to participate in GENERATION HD1 between January 2019 and April 2020—a recruitment rate nearly twice as fast as the average speed of large-scale clinical trials, and four times faster than Roche’s average recruitment rates for rare disease trials in the ten years preceding tominersen [ 8 ]. Additionally, increased site traffic was observed around major GENERATION HD1 study announcements on HDSA’s HD Trialfinder platform, a clinical trials matching service and educational resource for people affected by HD [ 9 ].…”

“…This group of global advocates led by HDSA and the Huntington Society of Canada (HSC) collaborates with pharmaceutical companies to make sure the voices of people affected by HD are represented in the design of clinical trials and educational materials [ 7 ]. HD-COPE was involved in the planning of GENERATION HD1 and has advised numerous pharmaceutical and biotech companies, providing insight on the realities of living with HD, input on patient-facing study materials, and recommendations related to trial design [ 8 ]. In the months since GENERATION HD1 and Wave’s PRECISION-HD studies drew to a close, members of HD-COPE as well as other community members and healthcare providers participated in a series of interviews with HDSA staff to reflect on their experiences.…”