Study 201
Study 201 showed that a ready-made mechlorethamine skin gel worked at least as well as a pharmacy-compounded ointment for early mycosis fungoides, and it was the main evidence for the US approval of Valchlor.
Overview
Study 201 was a randomised, multicentre, observer-blinded phase 2 pivotal trial of topical mechlorethamine in adults with stage IA to IIA mycosis fungoides-type cutaneous T-cell lymphoma who had received prior skin-directed therapy. Two hundred and sixty patients were assigned to mechlorethamine 0.02% gel or to mechlorethamine 0.02% compounded ointment, each applied once daily for up to 12 months, with tumour response and adverse events assessed monthly for six months and then every two months. The primary endpoint was the ratio of confirmed Composite Assessment of Index Lesion Severity (CAILS) response rates, with noninferiority predefined.
CAILS response was 58.5 percent with the gel and 47.7 percent with the ointment (ratio 1.23, 95% CI 0.97 to 1.55), which met the prespecified noninferiority criterion. Modified Severity-Weighted Assessment Tool response was 46.9 percent against 46.2 percent. Time-to-response analyses favoured the gel (JAMA Dermatology 2013). The registry results post 76 and 62 CAILS responders out of 130 patients per arm, the same 58.5 and 47.7 percent.
The US Valchlor label describes the same 260-patient study, notes that Valchlor 0.016 percent is equivalent to 0.02 percent mechlorethamine hydrochloride, excludes 18 patients with site randomisation violations from the efficacy table (119 versus 123), and reports CAILS response 60 percent against 48 percent (ratio 1.24, 95% CI 0.98 to 1.58). The FDA approved Valchlor in 2013 for stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma after skin-directed therapy. The registry records the study as completed under org id 2005NMMF-201-US.
- 58.5 vs 47.7 out of 100 had their tumour shrink with Mechlorethamine 0.02% gel compared with Mechlorethamine 0.02% compounded ointment; 10.8 more per 100.
- Roughly one extra person helped for every 9 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
- 46.9 vs 46.2 out of 100 had their tumour shrink with Mechlorethamine 0.02% gel compared with Mechlorethamine 0.02% compounded ointment; 0.7 more per 100.
- Roughly one extra person helped for every 143 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- These results apply to the people the trial enrolled: Stage IA to IIA mycosis fungoides-type cutaneous T-cell lymphoma after prior skin-directed therapy: once-daily mechlorethamine 0.02% gel compared with mechlorethamine 0.02% compounded ointment for up to 12 months. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
260 enrolled.
Ratio of gel to ointment response rates 1.23 (95% CI 0.97 to 1.55); noninferiority met; 130 per arm on the registry
SourceSimilar pages
not linked directly; found by shared links- CompanyRecordati
Shares Mechlorethamine (chlormethine), Helsinn.