Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

Phase 2

Completed

• Conditions: Mycosis Fungoides
• Interventions: Drug: mechlorethamine-MCH (nitrogen mustard)

• Registration Number: NCT00168064
• Lead Sponsor: Yaupon Therapeutics

Brief Summary

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).

Detailed Description

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.

Study Timeline

• Study First Submitted: 2005-09-07
• Study First Submitted QC: 2005-09-07
• Study First Posted: 2005-09-14
• Study Start: 2006-05
• Primary Completion: 2010-08
• Results First Submitted: 2011-07-12
• Study Completion: 2011-08
• Status Verified: 2012-10
• Results First Submitted QC: 2012-10-02
• Last Update Submitted: 2012-10-02
• Results First Posted: 2012-10-31
• Last Update Posted: 2012-10-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 260

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1 (PG - NM (MCH) 0.02%)	mechlorethamine-MCH (nitrogen mustard)	PG - mechlorethamine-MCH (nitrogen mustard) 0.02% gel To evaluate the tolerability and safety of topical mechlorethamine-MCH (nitrogen mustard) 0.02% ointment formulations in patients with stage I or IIA MF
2 (AP - MCH(NM) 0.02%)	mechlorethamine-MCH (nitrogen mustard)	AP - mechlorethamine-MCH (nitrogen mustard) 0.02% compounded in Aquaphor To evaluate the tolerability and safety of mechlorethamine-MCH (nitrogen mustard)0.02% ointment formulations in patients with stage I or IIA MF

Primary Outcome Measures

Name	Time	Method
Ratio of Response Rates Based on CAILS	Assessment made at Day 1 and every subsequent visit during treatment	The ratio of the response rate of the patients treated with the PG formulation to the response rate of the patients treated with the AP formulation. Skin response determined by at least a 50% reduction from baseline in the Composite Assessment of Index Lesion Severity (CAILS) following up to 12 months of treatment

Secondary Outcome Measures

Name	Time	Method
Severity-weighted Assessment Tool (SWAT) Within up to 12 Months by 2 or More Consecutive Observations Over at Least 4 Weeks	Assessment made at Day 1 and every subsequent visit during treatment
Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)	Baseline to end of therapy	Assessment of lesion distribution and severity. A responder analysis was performed on whether subject achieved at least 50% improvement on scale. This had to be confirmed on at least one visit at least 4 weeks apart.

Trial Locations

Locations (12)

University of Texas, Southwestern Medical Center; 🇺🇸 Dallas, Texas, United States

Northwestern University - Dept. of Dermatology; 🇺🇸 Chicago, Illinois, United States

Fox Chase Cancer Center; 🇺🇸 Philadelphia, Pennsylvania, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

NYU Medical Center Dept. of Dermatology; 🇺🇸 New York, New York, United States

Columbia University, Dept. of Dermatology; 🇺🇸 New York, New York, United States

Stanford University Medical Center; 🇺🇸 Stanford, California, United States

Oklahoma University; 🇺🇸 Tulsa, Oklahoma, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

The University of Texas, M.D. Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

Utah Clinical Trials, LLC; 🇺🇸 Salt Lake City, Utah, United States

University of Wisconsin; 🇺🇸 Madison, Wisconsin, United States