A phase II Pivotal Trial to Evaluate the Safety and Efficacy of Nitrogen Mustard

描述（由申请人提供）： 此申请是一个竞争性的延续，目前提供两年 资助一项2期关键性试验，测试MF患者外用NM制剂的安全性和有效性。 MF是最常见的皮肤T细胞淋巴瘤类型。 NM是一种烷化剂，用于全身治疗淋巴瘤。 NM与凡士林或AP配伍可能是治疗MF安全有效的外用药物。 已经开发了含PG的软膏制剂作为重AP载体的替代品，重AP载体可能难以应用于皮肤并且可能损害患者依从性。 一项多中心、随机、双盲试验已经启动，以测试118名患者中0.02% NM PG软膏与0.02% NM AP软膏的安全性和有效性。预计应计制将在第二年完成，但评估将持续到第三年并在第三年完成。 既往未接受过局部NM或卡莫司汀（BCNU）治疗的Ia、Ib或IIa期MF确诊患者符合资格。主要和次要疗效终点分别为索引病变严重程度的综合评估和严重程度加权评估工具。患者将研究软膏涂抹于受影响的皮肤区域（病变）或整个皮肤表面（取决于分期），每天一次，持续长达12个月。 应用的频率可以根据毒性进行调整。从第1个月至第6个月每4周评估一次肿瘤反应和毒性，从第7个月至第12个月每8周评估一次。 将在延长的12个月随访中采集其他毒性数据（鳞状细胞癌）。

DESCRIPTION (provided by applicant): This application is for a competitive continuation which currently provides two-years funding for a Phase 2, pivotal trial testing the safety and efficacy of topical NM formulations in patients with MF. MF is the most common type of cutaneous T-cell lymphoma. NM is an alkylating agent, used in the systemic treatment of lymphomas. NM compounded into petrolatum or AP may be a safe and effective topical treatment of MF. A PG-containing ointment formulation has been developed as an alternative to the heavy AP vehicle which can be difficult to apply to the skin and can compromise patient compliance. A multi-center, randomized, observer-blind trial has been initiated to test the safety and efficacy of a manufactured 0.02% NM PG ointment versus a compounded 0.02% NM AP ointment in 118 patients. It is anticipated that accrual will be completed by year two but assessment will continue into and be completed by year three. Established patients with Stage Ia, Ib or IIa MF, with no prior topical NM or carmustine (BCNU) therapy, are eligible. Primary and secondary efficacy endpoints are the Composite Assessment of Index Lesion Severity and Severity Weighted Assessment Tool, respectively. Patients apply study ointment to affected skin areas (lesions) or total skin surface (depending on stage) once daily for up to 12 months. The frequency of application can be adjusted for toxicity. Tumor response and toxicity are assessed every 4 weeks from months 1 to 6 and every 8 weeks from months 7 to12. Additional toxicity data (squamous cell carcinoma) will be captured in an extended 12-month follow-up.