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Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Yaupon Therapeutics
ClinicalTrials.gov Identifier:
NCT00168064
First received: September 7, 2005
Last updated: October 2, 2012
Last verified: October 2012
History of Changes
  Purpose

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).


Condition Intervention Phase
Mycosis Fungoides
 Drug: mechlorethamine-MCH (nitrogen mustard)
 Phase 2

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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Yaupon Therapeutics:

Primary Outcome Measures:
  • Ratio of Response Rates Based on CAILS [ Time Frame: Assessment made at Day 1 and every subsequent visit during treatment ] [ Designated as safety issue: No ]
    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:
  • Severity-weighted Assessment Tool (SWAT) Within up to 12 Months by 2 or More Consecutive Observations Over at Least 4 Weeks [ Time Frame: Assessment made at Day 1 and every subsequent visit during treatment ] [ Designated as safety issue: Yes ]
  • Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT) [ Time Frame: Baseline to end of therapy ] [ Designated as safety issue: No ]
    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.


Enrollment: 260
Study Start Date: May 2006
Study Completion Date: August 2011
Primary Completion Date: August 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1 (PG - NM (MCH) 0.02%)
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
 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
Other Names:
  • mechlorethamine
  • nitrogen mustard
Active Comparator: 2 (AP - MCH(NM) 0.02%)
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
 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
Other Names:
  • mechlorethamine
  • nitrogen mustard

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00168064

Locations
United States, California
Stanford University Medical Center
Stanford, California, United States, 94305
United States, Illinois
Northwestern University - Dept. of Dermatology
Chicago, Illinois, United States, 61611
United States, New York
NYU Medical Center Dept. of Dermatology
New York, New York, United States, 10016
Columbia University, Dept. of Dermatology
New York, New York, United States, 10032
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Oklahoma
Oklahoma University
Tulsa, Oklahoma, United States, 74104
United States, Pennsylvania
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Fox Chase Cancer Center
Philadelphia, Pennsylvania, United States, 19111-2497
United States, Texas
University of Texas, Southwestern Medical Center
Dallas, Texas, United States, 75390
The University of Texas, M.D. Anderson Cancer Center
Houston, Texas, United States, 77030
United States, Utah
Utah Clinical Trials, LLC
Salt Lake City, Utah, United States, 84107
United States, Wisconsin
University of Wisconsin
Madison, Wisconsin, United States, 53791
Sponsors and Collaborators
Yaupon Therapeutics
Investigators
Study Director: Stuart Lessin, M.D. Fox Chase Cancer Center
  More Information

Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Yaupon Therapeutics
ClinicalTrials.gov Identifier: NCT00168064     History of Changes
Other Study ID Numbers: 2005NMMF-201-US
Study First Received: September 7, 2005
Results First Received: July 12, 2011
Last Updated: October 2, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Yaupon Therapeutics:
Mycosis Fungoides
Nitrogen Mustard
Cutaneous T-Cell Lymphoma
CTCL - Mycosis Fungoides

Additional relevant MeSH terms:
Mycoses
Mycosis Fungoides
Lymphoma, T-Cell, Cutaneous
Lymphoma, T-Cell
Lymphoma, Non-Hodgkin
Lymphoma
Neoplasms by Histologic Type
Neoplasms
Lymphoproliferative Disorders
Lymphatic Diseases
 Immunoproliferative Disorders
Immune System Diseases
Mechlorethamine
Nitrogen Mustard Compounds
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on June 18, 2013