Topical chemotherapy in cutaneous T-cell lymphoma: positive results of a randomized, controlled, multicenter trial testing the efficacy and safety of a novel mechlorethamine, 0.02%, gel in mycosis fungoides
The primary report of Study 201: a ready-made mechlorethamine gel cleared index lesions in 58.5 percent of early mycosis fungoides patients against 47.7 percent with the traditional compounded ointment, meeting the noninferiority bar.
Overview
Randomised, controlled, observer-blinded, multicentre trial at academic medical and cancer centres comparing mechlorethamine hydrochloride 0.02% gel with mechlorethamine 0.02% compounded ointment, applied once daily for up to 12 months, in 260 patients with stage IA to IIA mycosis fungoides who had not used topical mechlorethamine within 2 years and were naive to topical carmustine. The primary endpoint was response by the Composite Assessment of Index Lesion Severity (CAILS); secondary endpoints were the Modified Severity-Weighted Assessment Tool and time-to-response analyses.
Response rates for gel versus ointment were 58.5 versus 47.7 percent by CAILS and 46.9 versus 46.2 percent by the Modified Severity-Weighted Assessment Tool. The ratio of gel to ointment CAILS response rates was 1.23 (95% CI 0.97 to 1.55), which met the prespecified criterion for noninferiority. Time-to-response analyses demonstrated superiority of the gel.
- CAILS response 58.5% with mechlorethamine 0.02% gel vs 47.7% with compounded ointment; ratio 1.23 (95% CI 0.97 to 1.55), noninferiority met.
- Modified Severity-Weighted Assessment Tool response 46.9% vs 46.2%.
- Time-to-response analyses favoured the gel.
This trial gave mechlorethamine, the first chemotherapy drug ever used, a modern licensed form: the FDA approved Valchlor gel in 2013 for early mycosis fungoides-type cutaneous T-cell lymphoma after skin-directed therapy, replacing pharmacy-compounded preparations of uncertain stability.
- Active-controlled noninferiority design against a compounded ointment; no placebo or untreated arm.
- Observer-blinded rather than double-blinded for the applied product.
- The label's efficacy table excludes 18 patients with randomisation protocol violations and reports 60% vs 48%.
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Shares Mechlorethamine (chlormethine), Helsinn.