511 Analysis of Clinical Outcome Assessments in Clinical Trials for Huntington’s Disease

Abstract

OBJECTIVES/GOALS: Examine the use of Patient-Reported Outcomes (PRO) in Huntington’s Disease (HD) clinical trials (CT) and compare across time and sponsor types. METHODS/STUDY POPULATION: Conduct literature review on 1. background of HD, 2. what symptoms and outcome measures are most important to patients, including the Patient-Focused Drug Development (PFDD) meeting for HD–led by the U.S. Food and Drug Administration (FDA), 3. what outcome measure tools currently exist and what they measure. Utilizing Clinicaltrials.gov, trials for HD were examined to assess the number of trials conducted, what COAs were used, and funding types. Trials were filtered by study type (keep Interventional) and status (filter out suspended, terminated, unknown, and withdrawn). The frequency of COAs will then be mapped based on the symptoms from the PFDD meeting. RESULTS/ANTICIPATED RESULTS: From the PFDD meeting for HD, symptoms that were important to patients include cognitive impairment, depression and anxiety, and motor symptoms. From the 139 interventional studies that were active, complete, recruiting, or not recruiting, 79 studies were conducted by Industry, 3 by NIH, 93 by Other (Academia/Community Organizations), and 1 by a U.S. Federal Agency (other than NIH). One of the most commonly used COA is the Unified Huntington’s Disease Rating Scale (UHDRS), which includes a motor, cognitive, and behavior assessment, and an assessment on functional capacity and independence. Of the 27 out of 139 trials analyzed to date, there were a total of 37 COAs. DISCUSSION/SIGNIFICANCE: The widespread use of UHDRS can be attributed to its standardization in 1999. It captures the symptoms of HD that are most important to patients. Because UHDRS is not sensitive to any one symptom, other COAs have been developed which focus on unique aspects of HD and allow for its earlier detection.