Poster 4: The Functional Rating Scale Taskforce for Pre-Huntington's Disease: An Empirically-Driven Initiative for New Scale Development

Abstract

There is an unmet need for the development of treatments for Huntington's disease (HD). The ideal goal would be to develop drugs that can be administered sufficiently early (i.e., pre-HD), such that the full manifestations never develop or are at least delayed. Unfortunately, there is currently no sensitive validated tool capable of measuring symptomatic changes in pre-HD that can be used in clinical trials. The Functional Rating Scale Taskforce for pre-Huntington Disease (FuRST-pHD) is a multinational, multidisciplinary initiative with the goal to develop a data-driven, comprehensive, psychometrically sound, sensitive, functional rating scale to be utilized in clinical trials to measure symptoms in patients at the pre-HD stage of the disease. The Taskforce will first identify signs and symptom clusters that should be assessed in pre-HD patients, based on input from a variety of sources, including clinical experts, patients, caregivers, and regulatory agencies. The Taskforce will then develop clinical measures of these signs and symptoms using an iterative processes involving field testing and psychometric analyses. The Taskforce will also rely on the analyses of existing datasets to identify items that may have utility in measuring pre-HD symptomatology. The Taskforce is currently analyzing data from PREDICT-HD, using item response theory (IRT), an approach that has been shown to be a powerful method for evaluating the performance of individual items of rating scales by assessing the relationship between item scores and severity. The development of a new data-driven, reliable, valid, and easily administered instrument would be valuable in the assessment of pre-HD symptoms in clinical trials. This collaborative effort welcomes input from HD researchers. FuRST-pHD is sponsored by CHDI Foundation.