Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

Mycosis Fungoides Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00168064
• Other study ID #: 2005NMMF-201-US
• Status: Completed
• Phase: Phase 2
• First received: September 7, 2005
• Last updated: October 2, 2012
• Start date: May 2006
• Est. completion date: August 2011

Study information

• Verified date: October 2012
• Source: Yaupon Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 260
• Est. completion date: August 2011
• Est. primary completion date: August 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mycoses
• Mycosis Fungoides

Intervention

Drug:
• mechlorethamine-MCH (nitrogen mustard)
All affected areas (lesions) are to be treated once daily for twelve months with mechlorethamine-MCH (nitrogen mustard) 0.02% PG or NM 0.02% AP ointment

Locations

Country	Name	City	State
United States	Northwestern University - Dept. of Dermatology	Chicago	Illinois
United States	University of Texas, Southwestern Medical Center	Dallas	Texas
United States	Duke University Medical Center	Durham	North Carolina
United States	The University of Texas, M.D. Anderson Cancer Center	Houston	Texas
United States	University of Wisconsin	Madison	Wisconsin
United States	Columbia University, Dept. of Dermatology	New York	New York
United States	NYU Medical Center Dept. of Dermatology	New York	New York
United States	Fox Chase Cancer Center	Philadelphia	Pennsylvania
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Utah Clinical Trials, LLC	Salt Lake City	Utah
United States	Stanford University Medical Center	Stanford	California
United States	Oklahoma University	Tulsa	Oklahoma

Sponsors (1)

Lead Sponsor	Collaborator
Yaupon Therapeutics

Country where clinical trial is conducted

United States, 

References & Publications (3)

Kim YH, Jensen RA, Watanabe GL, Varghese A, Hoppe RT. Clinical stage IA (limited patch and plaque) mycosis fungoides. A long-term outcome analysis. Arch Dermatol. 1996 Nov;132(11):1309-13. — View Citation

Kim YH, Martinez G, Varghese A, Hoppe RT. Topical nitrogen mustard in the management of mycosis fungoides: update of the Stanford experience. Arch Dermatol. 2003 Feb;139(2):165-73. — View Citation

Kim YH. Management with topical nitrogen mustard in mycosis fungoides. Dermatol Ther. 2003;16(4):288-98. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Ratio of Response Rates Based on CAILS	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	Assessment made at Day 1 and every subsequent visit during treatment	No
Secondary	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	Yes
Secondary	Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)	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.	Baseline to end of therapy	No