A narrative review of phase III and IV clinical trials for the pharmacological treatment of Huntington's disease in adults

对成人亨廷顿病药物治疗的III期和IV期临床试验进行叙述性综述

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Abstract

Huntington disease (HD) is a hereditary neurodegenerative condition characterized by progression of motor, cognitive, and psychiatric abnormalities. Despite the lack of definitive medications, current research efforts are actively assessing novel pharmaceutical interventions through phase III and IV clinical trials to mitigate the limited effectiveness of existing therapeutic approaches. The primary objective of these trials is to enhance symptom management and improve the overall quality of life for individuals diagnosed with HD. These trials show potential for development of further efficacious therapeutic interventions in future. To identify and provide details about medications tested in completed phase III and IV clinical trials for managing HD in adults. Publicly available and relevant phase III and IV trials registered at ClinicalTrials.gov analyzed. Usage of the trialed medications for HD reviewed. As of November 10, 2023, there were 242 phase III and IV clinical trials related to HD. Eight clinical trials from these met the inclusion criteria for the current study. The medications used in phase III and IV trials are minocycline, valbenazine, deutetrabenazine, tominersen, pridopidine (phase III), and memantine (phase IV). Evaluating phase III and IV clinical studies on HD highlights the importance of tailored approaches for each patient's unique disease presentation. Current medications aim to manage HD symptoms, potentially improving outcomes and reducing disease progression risks. The growing emphasis on specific approaches reflects a better understanding of HD's diverse symptoms, presenting opportunities for more effective and personalized treatment strategies.

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