SAFETY AND PHARMACOKINETICS STUDY OF ARIMOCLOMOL IN ALS

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这是一项IIA期双盲研究，旨在评估3种剂量阿利莫洛尔的安全性和脑脊液渗透情况。来自多个中心的80名受试者将被随机分为4组：安慰剂或阿利莫洛尔组，每日25、50或100 mg，并接受为期12周的药物治疗。治疗4周后，受试者将进入GCRC接受血液和脑脊液药代动力学(PK)研究。每种剂量都将根据安全性和PK数据进行评估，并将为IIb阶段研究选择最佳的两种剂量。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is a phase IIA double-blind study to assess the safety and CSF penetration of 3 doses of Arimoclomol. Eighty subjects from multiple centers will be randomized to 1 of 4 groups: placebo or Arimoclomol at 25, 50, or 100mg t.i.d., and receive medication for 12 weeks. After 4 weeks of treatment, subjects will be admitted to the GCRC for a blood and CSF pharmacokinetic (PK) study. Each dose will be assessed based on the safety and the PK data and the best two dosages for the phase IIb study will be selected.