Evaluate the efficacy & safety of a novel 0.02% mechlorethamine (MCH) gel in mycosis fungoides (MF).
Multi-center, randomized, observer-blinded, active controlled trial, comparing 0.02% MCH gel with 0.02% MCH compounded ointment. MCH was applied once daily for up to 12 months. Tumor response and adverse events (AEs) were assessed every month (months 1–6) and every 2 months (months 7–12). Serum drug levels were drawn on a subset of subjects.
Academic medical and/or cancer centers
260 Stage IA-IIA MF patients who had not used topical MCH within 2 years and were naïve to prior use of topical carmustine therapy.
Main Outcome Measures
Response rates (RRs) of all subjects based on primary (composite assessment of index lesion severity - CAILS) and secondary (modified severity weighted assessment tool (mSWAT) and time to response analyses) clinical endpoints.
RR for MCH gel and ointment were 59% vs. 48% by CAILS and 46.9% vs. 46.2% by mSWAT, respectively. The ratio of CAILS RR of MCH gel to ointment was 1.23 (CI: 0.97–1.55), which met the pre-specified criterion for non-inferiority. Time to response demonstrated superiority of MCH gel to ointment (p<0.012). No drug-related serious AEs were seen. Twenty percent (20%) of enrolled patients on gel and 17% on ointment withdrew due to drug-related skin irritation. No systemic absorption of the study medication was detected.
These results demonstrate the safety and efficacy of a novel 0.02% MCH gel in the treatment of MF patients.
Clinical Trial Registration