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

点击翻译按钮获取中文摘要

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.