Evaluation of Efficacy and Mechs of Topical Thalidomide for cGVHD Stomatitis

2007年6月26日，FDA最终批准了沙利度胺局部凝胶IND 76,793的使用。受试者招募于2007年11月在美国国立卫生研究院临床中心开始。所有受试者均给予书面知情同意。所有数据都被收集起来，研究对象按照方案自行给药。外用沙利度胺和安慰剂由美国国立卫生研究院临床中心药物开发服务部准备。华盛顿州西雅图的弗雷德·哈钦森癌症研究中心(FHCRC)机构审查委员会于2008年8月批准了该方案。在首席调查员和高级研究护士于2009年1月进行现场启动访问后，FHCRC被添加为该方案的第二个临床地点。受试者的积累慢于预期，有10名受试者在NIH临床中心登记超过2年，没有受试者在FHCRC登记超过1年。因此，随着国家牙科和颅面研究机构审查委员会的批准，数据分析正在进行中，这项研究结束了主题应计。数据分析正在检查局部使用沙利度胺凝胶治疗口腔溃疡性慢性移植物抗宿主病的初步结果，唾液、口腔溃疡渗出液和血浆中促炎细胞因子的水平，以及口腔颊粘膜和口咽部疼痛的基因表达。

The FDA gave final clearance for use of thalidomide topical gel IND 76, 793, on June 26, 2007. Subject recruitment started in November 2007 at the Clinical Center, NIH. All subjects gave written informed consent. All data were collected, and study drug self-administered by subjects per protocol. Topical thalidomide and placebo was prepared by the Pharmaceutical Development Service, Clinical Center, NIH. The Fred Hutchinson Cancer Research Center's (FHCRC), Seattle, WA., institutional review board approved the protocol in August, 2008. Following the site initiation visit conducted by the principal investigator and senior research nurse in January 2009, the FHCRC was added as the second clinical site for this protocol. Subject accrual was slower than anticipated with 10 subjects enrolled at the Clincal Center, NIH, over 2 years, and no subjects enrolled at the FHCRC over 1 year. Therefore, the study was closed to subject accrual with data analyses ongoing with National Institute of Dental and Cranifacial Research Institutional Review Board, NIH, approval. Data analysis is examining preliminary findings of topical thalidomide gel on oral ulcerative chronic GVHD, levels of proinflammatory cytokines in saliva, oral ulcer exudate, and plasma, as well as gene expression in oral buccal mucosa, and oropharyngeal pain.