Current clinical trials of new therapeutic agents for Huntington's disease

Huntington's disease (HD) is a rare, inherited neurodegenerative disorder caused by a CAG repeat expansion mutation in the HTT gene. HD is characterized by a triad of motor, cognitive, and psychiatric symptoms, and typically progresses over 10–25 years before ultimately leading to complete disability and premature death. Despite extensive research, there is currently no approved disease-modifying therapy for HD. Clinical trials are a critical tool for evaluating new treatments for HD and improving patient outcomes. The clinicaltrials.gov website provides a comprehensive database of ongoing clinical trials in HD. This book chapter provides an overview of the current landscape of clinical trials in HD, based on data available in the clinicaltrials.gov database. It first briefly describes the different phases of clinical trials and the regulatory requirements for conducting trials in HD, and then reviews the current state of the most important clinical trials in HD, including available information on the study design, participant populations, specific intervention, and relevant outcome measures. Finally, this chapter briefly discusses conclusions and future directions highlighting the challenges and opportunities in designing and conducting clinical trials in HD and the need for continued collaboration and innovation in the field.