CLINICAL TRIAL: PEDIATRIC STUDY OF SODIUM PHENYLBUTYRATE W/TYPE II/III SPINAL MU

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。 建议的方案是在NPTUNE(NINDS试点治疗网络)项目下通过位于马里兰州罗克维尔的CRO Westat，Inc.通过NIH的分包进行的。该协议代表了NPTUNE 01协议的第一阶段，这是一项多中心的第一阶段临床试验，苯丁酸钠治疗一种称为脊肌萎缩症(SMA)的儿科神经肌肉疾病。苯丁酸钠是FDA批准用于尿素循环障碍的药物。NPTUNE 01适用于2至12岁的2型和3型SMA患者，这是一种不太严重的变种。第一阶段包括短期(1个月)治疗阶段，以确定II型和III型SMA患者的最大耐受剂量。这将作为MTD第二个长期(3个月)治疗阶段的基础，在新的一组患者中进行。作为第一阶段研究，重点将是记录剂量限制毒性、不良事件/反应和药代动力学。患者对药物方案的依从性也将得到特别考虑。

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. The proposed protocol is being conducted under the NPTUNE (NINDS Pilot Therapeutics Network) project through an NIH subcontract via the CRO Westat, Inc. in Rockville, MD. This protocol represents Stage 1 of Protocol NPTUNE 01, a multi-center, Phase I clinical trial of sodium phenylbutyrate in a pediatric neuromuscular disorder called spinal muscular atrophy (SMA). Sodium phenylbutyrate is FDA-approved for use in urea cycle disorders. NPTUNE 01 is for patients with Type 2 and 3 SMA, the less severe variants, between the ages of 2 and 12 years. Stage 1 consists of a short-term (1 month) treatment stage to determine the maximum tolerated dose of the medication in patients with Type II and III SMA. This will serve as a basis for a second long-term (3 months) treatment stage at the MTD, conducted in a new set of patients. As a Phase I study, the focus will be on documenting dose-limiting toxicities, adverse events/reactions, and pharmacokinetics. Patient compliance with the drug regimen will also be given special consideration.