Studies Of Hereditary Neurological Disease: Clinical Trials

遗传性神经系统疾病的研究：临床试验

• 批准号: 8158222
• 负责人: Kenneth Fischbeck
• 金额: $ 64.9万
• 依托单位: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/8158222
• 关键词: Clinical Trials; Inherited; nervous system disorder

The purpose of this research program is to develop safe and effective treatments for hereditary neurological disorders. Specific research accomplishments in the past year include the following: (1) further analysis of the results of a phase 2 study of high dose idebenone treatment for Friedreich's ataxia, (2) analysis of the results of a cross sectional study of spinal and bulbar muscular atrophy (SBMA, Kennedy's disease), and (3) completion of a phase 2 clinical trial to test the feasibility of dutasteride treatment for SBMA. The Friedreich's idebenone trial showed evidence of neurological efficacy with intermediate and high dose treatment in ambulatory subjects. The SBMA dutasteride trial also showed evidence of efficacy, although the primary outcome measure, quantitative muscle testing, did not show a significant benefit.

该研究计划的目的是为遗传神经系统疾病开发安全有效的治疗方法。过去一年中的具体研究成就包括：（1）进一步分析弗里德里希共济失调的高剂量易替酮治疗的结果2阶段研究的结果，（2）分析脊柱和球肌肉萎缩术的横截面研究结果（肯尼迪氏病SBMA，肯尼迪氏病），以及（3）对S阶段2阶段的临床临床临床临床审查的分析。 弗里德里希（Friedreich）的iDebenone试验显示了在门诊受试者中使用中等和高剂量治疗的神经系统疗效的证据。 SBMA Dutasteride试验还显示了功效的证据，尽管主要结局指标（定量肌肉测试）并未显示出显着的好处。