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Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

Primary Purpose

Mycosis Fungoides

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
mechlorethamine-MCH (nitrogen mustard)
Sponsored by
Yaupon Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mycosis Fungoides focused on measuring Mycosis Fungoides, Nitrogen Mustard, Cutaneous T-Cell Lymphoma, CTCL - Mycosis Fungoides

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Stanford University Medical Center
  • Northwestern University - Dept. of Dermatology
  • NYU Medical Center Dept. of Dermatology
  • Columbia University, Dept. of Dermatology
  • Duke University Medical Center
  • Oklahoma University
  • University of Pennsylvania
  • Fox Chase Cancer Center
  • University of Texas, Southwestern Medical Center
  • The University of Texas, M.D. Anderson Cancer Center
  • Utah Clinical Trials, LLC
  • University of Wisconsin

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

1 (PG - NM (MCH) 0.02%)

2 (AP - MCH(NM) 0.02%)

Arm Description

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

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Severity-weighted Assessment Tool (SWAT) Within up to 12 Months by 2 or More Consecutive Observations Over at Least 4 Weeks
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.

Full Information

First Posted
September 7, 2005
Last Updated
October 2, 2012
Sponsor
Yaupon Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT00168064
Brief Title
Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides
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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2012
Overall Recruitment Status
Completed
Study Start Date
May 2006 (undefined)
Primary Completion Date
August 2010 (Actual)
Study Completion Date
August 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Yaupon Therapeutics

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mycosis Fungoides
Keywords
Mycosis Fungoides, Nitrogen Mustard, Cutaneous T-Cell Lymphoma, CTCL - Mycosis Fungoides

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
260 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1 (PG - NM (MCH) 0.02%)
Arm Type
Active Comparator
Arm Description
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
Arm Title
2 (AP - MCH(NM) 0.02%)
Arm Type
Active Comparator
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
mechlorethamine-MCH (nitrogen mustard)
Other Intervention Name(s)
mechlorethamine, nitrogen mustard
Intervention Description
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
Primary Outcome Measure Information:
Title
Ratio of Response Rates Based on CAILS
Description
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
Time Frame
Assessment made at Day 1 and every subsequent visit during treatment
Secondary Outcome Measure Information:
Title
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
Title
Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)
Description
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.
Time Frame
Baseline to end of therapy

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stuart Lessin, M.D.
Organizational Affiliation
Fox Chase Cancer Center
Official's Role
Study Director
Facility Information:
Facility Name
Stanford University Medical Center
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Northwestern University - Dept. of Dermatology
City
Chicago
State/Province
Illinois
ZIP/Postal Code
61611
Country
United States
Facility Name
NYU Medical Center Dept. of Dermatology
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Columbia University, Dept. of Dermatology
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Oklahoma University
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74104
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Fox Chase Cancer Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19111-2497
Country
United States
Facility Name
University of Texas, Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
The University of Texas, M.D. Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Utah Clinical Trials, LLC
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Facility Name
University of Wisconsin
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53791
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
12588222
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. doi: 10.1001/archderm.139.2.165.
Results Reference
background
PubMed Identifier
14686971
Citation
Kim YH. Management with topical nitrogen mustard in mycosis fungoides. Dermatol Ther. 2003;16(4):288-98. doi: 10.1111/j.1396-0296.2003.01640.x.
Results Reference
background
PubMed Identifier
8915308
Citation
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.
Results Reference
background
PubMed Identifier
35536441
Citation
Querfeld C, Scarisbrick JJ, Assaf C, Kim YH, Guitart J, Quaglino P, Hodak E. Chlormethine Gel Versus Chlormethine Ointment for Treatment of Patients with Mycosis Fungoides: A Post-Hoc Analysis of Clinical Trial Data. Am J Clin Dermatol. 2022 Jul;23(4):561-570. doi: 10.1007/s40257-022-00687-y. Epub 2022 May 10.
Results Reference
derived
PubMed Identifier
34091453
Citation
Querfeld C, Scarisbrick JJ, Assaf C, Guenova E, Bagot M, Ortiz-Romero PL, Quaglino P, Bonizzoni E, Hodak E. Post hoc Analysis of a Randomized, Controlled, Phase 2 Study to Assess Response Rates with Chlormethine/Mechlorethamine Gel in Patients with Stage IA-IIA Mycosis Fungoides. Dermatology. 2022;238(2):347-357. doi: 10.1159/000516138. Epub 2021 Jun 4.
Results Reference
derived
PubMed Identifier
23069814
Citation
Lessin SR, Duvic M, Guitart J, Pandya AG, Strober BE, Olsen EA, Hull CM, Knobler EH, Rook AH, Kim EJ, Naylor MF, Adelson DM, Kimball AB, Wood GS, Sundram U, Wu H, Kim YH. 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. JAMA Dermatol. 2013 Jan;149(1):25-32. doi: 10.1001/2013.jamadermatol.541.
Results Reference
derived

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Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides

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