# Study 201

Source: https://onco.cc/trials/study-201/  
OnCo record `study-201` (Trial). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Trial
- Status: positive
- Last checked: 2026-09-24
- Also known as: 2005NMMF-201-US; Valchlor pivotal trial
- Registry id: NCT00168064
- Phase: 2
- Setting: 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
- Sponsor: Yaupon Therapeutics
- Enrolled: 260
- Result: CAILS response 58.5% with mechlorethamine gel against 47.7% with compounded ointment (ratio 1.23, met noninferiority); modified SWAT response 46.9% against 46.2%.
- Outcomes: CAILS response rate: Mechlorethamine 0.02% gel 58.5% vs Mechlorethamine 0.02% compounded ointment 47.7%; Modified SWAT response rate: Mechlorethamine 0.02% gel 46.9% vs Mechlorethamine 0.02% compounded ointment 46.2%

## Sources

- ClinicalTrials.gov NCT00168064: https://clinicaltrials.gov/study/NCT00168064
- JAMA Dermatology 2013: https://doi.org/10.1001/2013.jamadermatol.541
- Valchlor label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6a52e4c2-6a9f-4ebb-bc77-046b4f8bcd57

## Connected records

- cancers: [Cutaneous T-cell lymphoma (mycosis fungoides and Sezary syndrome)](https://onco.cc/cancers/cutaneous-t-cell-lymphoma/)
- technologies: [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/)
- drugs: [Mechlorethamine (chlormethine)](https://onco.cc/drugs/mechlorethamine/)
- companies: [Helsinn](https://onco.cc/companies/helsinn/)
- key papers: [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](https://onco.cc/key-papers/paper-study-201-jama-dermatol-2013/)

---
JSON: https://onco.cc/api/v1/entities/study-201.json