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外用凝胶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。收集所有数据，并研究受试者根据方案自行给药。局部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处制备。弗雷德哈钦森癌症研究中心（FHCRC），西雅图，华盛顿州。2008年8月，美国机构审查委员会批准了该议定书。在首席研究员和高级研究护士于2009年1月进行现场启动访问之后，FHCRC被添加为该方案的第二个临床站点。受试者累积比预期的要慢，在NIH临床中心注册的10名受试者超过2年，在FHCRC注册的受试者超过1年。因此，在美国国立牙科和颅面研究机构审查委员会（NIH）的批准下，该研究结束了受试者应计性数据分析。数据分析检查了局部沙利度胺凝胶对口腔溃疡性慢性GVHD、唾液、口腔溃疡渗出液和血浆中促炎细胞因子水平以及口咽疼痛的初步发现。

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 examined 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 oropharyngeal pain.