Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

A Phase II Pivotal Trial to Evaluate the Safety and Efficacy of Nitrogen Mustard (NM) 0.02% Ointment Formulations in Patients With Stage I or IIA Mycosis Fungoides (MF)

This study will evaluate the efficacy, tolerability and safety of the topical application of mechlorethamine (MCH) formulations in patients with stage I or IIA mycosis fungoides (MF).

The successful use of mechlorethamine (MCH) as a topical agent in the treatment of mycosis fungoides, a form of cutaneous T-cell lymphoma, was first reported in the late 1950s, and provided a rationale for skin-directed chemotherapy that minimized systemic toxicity. Since then, multiple investigators have demonstrated the safety and efficacy of topically applied MCH in the treatment of mycosis fungoides. This study will evaluate the efficacy (non-inferiority), tolerability and safety of topical application of MCH proprietary gel versus a compounded ointment formulation in Aquaphor in patients with stage I or IIA MF.

Inclusion Criteria:

• Patients with mycosis fungoides confirmed by a skin biopsy
• Stage I or IIA patients must have been treated previously with prior topical therapies including PUVA, UVB, topical steroids, but not NM within the past 2 years, or topical carmustine (BCNU)
• Patients must be otherwise healthy with acceptable organ function.
• Prior to initiating study therapy, patients must not have had topical therapy within four weeks
• Lab values within normal range
• Willing/able to give consent
• Must use effective means of contraception if of childbearing potential

Exclusion Criteria:

• Newly diagnosed mycosis fungoides with no prior therapy
• A prior history of treatment with topical NM within the past 2 years or topical carmustine (BCNU)
• Use of topical or systemic therapies for MF within four (4) weeks of entry in the study
• Patients with a diagnosis of stage IIB-IV MF
• Serious known concurrent medical illness or infection, which could potentially present a safety risk and/or prevent compliance with the requirements of the treatment program
• Pregnant or nursing females, or males and females of childbearing potential, not using an effective means of contraception
• Patients who have had radiation therapy within one year of study start
• Patients who have a history of a higher T score than T2 or a higher N score than N1
• Patients who do not agree to do all labs at one site