Phase ll Study NH001 to Treat Severe Traumatic Brain Injury Pts in a Vegetative

NH001 治疗植物人严重创伤性脑损伤患者的 II 期研究

• 批准号: 7366872
• 负责人: ELKAN GAMZU
• 金额: $ 34.54万
• 依托单位: NEUROHEALING PHARMACEUTICALS, INC.
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-07-01 至 2012-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7366872
• 关键词: Phase; ll; Study; NH001; Treat

DESCRIPTION (provided by applicant): Each year 1.4 million Americans sustain a TBI. Of these patients, 50,000 people die and 235,000 people are hospitalized and survive. The survivors often remain in a state of altered consciousness (coma, VS or MCS) for an extended period of time. These states of altered consciousness are characterized by the lack of awareness and lack of interaction with the environment. Patients that remain in a VS or a MCS for one month after a TBI, have a poor prognosis. Thirty-three percent die, 43% remain in a vegetative state or are severely disabled and only 24% make a moderate or good recovery. Dopamine agonists have been shown to help in regaining consciousness and facilitating rehabilitation. They induce awareness, accelerate the recovery and improve the functional outcome of post-TBl coma VS and MCS patients. In 2006, NeuroHealing Pharmaceuticals received an orphan drug designation for NH001 for the treatment of patients in a VS or MCS. Apomorphine is a FDA approved drug for the treatment of Parkinson's disease. It is a more powerful dopamine agonist than any of the dopamine agents previously tried in post-TBI unconscious patients. It is administered subcutaneously using a portable infusion pump for 12 hours per day. A steady high level of dopamine stimulation is achieved during the diurnal hours and the patient is allowed to rest during the night. NeuroHealing is currently conducting a 10-patient open-label clinical study of NH001 to test this hypothesis. Six patients have been treated thus far. All have regained consciousness, recovered their ability to follow commands, and their capacity to participate in active physical and cognitive rehabilitation. Three patients have been followed for over one year with no lapses back into unconsciousness. The investigators propose to conduct a clinical study to evaluate the hypothesis that patients treated with NH001 will experience a more rapid return to consciousness with better functional outcomes than patients receiving placebo.

描述（由申请人提供）： 每年有140万美国人维持TBI。 在这些患者中，有50,000人死亡，235,000人住院并存活。幸存者通常会长时间保持意识改变（昏迷，VS或MC）的状态。这些改变意识的状态的特征是缺乏意识和与环境的相互作用。 TBI后保留在VS或MCS中一个月的患者的预后较差。 33％的死亡，有43％的人处于植物状态或严重残疾，只有24％的人进行中等或良好的康复。 多巴胺激动剂已被证明有助于恢复意识和促进康复。它们引起意识，加速恢复并改善TBL COMA与MCS患者的功能结果。 2006年，Neurohealing Pharmaceuticals接受了NH001的孤儿药物名称，用于在VS或MC中治疗患者。 阿哌丁碱是FDA批准的药物，用于治疗帕金森氏病。 与以前在TBI失去知觉的患者中尝试过的任何多巴胺药物相比，这是一种更强大的多巴胺激动剂。 它是使用便携式输液泵皮下施用的，每天12小时。 在昼夜小时内实现了稳定的高水平多巴胺刺激，并允许患者在夜间休息。 Neurohealing目前正在对NH001进行10名患者开放标签临床研究，以检验该假设。 到目前为止，已有六名患者接受治疗。所有人都恢复了意识，恢复了遵循命令的能力，并能够参与积极的身体和认知康复。 三名患者被遵循了一年以上，没有失误恢复到无意识。 研究人员建议进行一项临床研究，以评估接受NH001治疗的患者的假设将比接受安慰剂的患者更快地回报意识。