The number of novel agents for potential symptomatic and disease modifying treatments of Huntington Disease (HD) is increasing rapidly. Treatment priorities for investigators, sponsors or regulators may differ from those for individuals with HD and families. We know a great deal about HD from clinician and scientist reports, but very little data have been systematically collected directly from persons affected by HD.
To address this gap, the Huntington Study Group (HSG) is developing an online, observational study platform, myHDstory™. A critical first step for myHDstory™ is the pilot study, Making HD Voices Heard. The pilot study will ask people who are diagnosed with HD to report what they experience and how they function. The myHDstory™ platform was developed in collaboration with technology companies Grey Matter Technologies (GMT) and NeuroTargeting (NT).
Making HD Voices Heard is being conducted to help improve understanding of HD — for example, how individuals with HD experience their symptoms, and how the disease affects overall health and personal well-being. In addition, the information collected in this study will help to create better tools for measuring benefit in HD clinical trials. For those participants who have undergone genetic testing for HD, we are also interested in learning CAG test results and what the genetic testing experience was like for them. Participation in Making HD Voices Heard is voluntary and free. No medical treatments or medical advice will be provided as part of this study.
We will use this pilot study to better plan the next studies of the myHDstory™ project. myHDstory will provide a means for others from HD families (presymptomatic, gene negative, at-risk, caregivers) to participate in research studies in the future. It will also be a means for clinical trial participants to contribute data between study visits and to be followed long-term after conclusion of an interventional study. The platform will serve as a rapid mechanism for testing of new clinical trial endpoints and other research methodology.
