Clinical trial NCT00168064: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Mycosis Fungoides
Keywords: Mycosis Fungoides
Nitrogen Mustard
Cutaneous T-Cell Lymphoma
CTCL - Mycosis Fungoides
Lead sponsor: Yaupon Therapeutics
Sponsor class: INDUSTRY
First posted: 2005-09-14
Enrollment: 260
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Ratio of Response Rates Based on CAILS
timeFrame: Assessment made at Day 1 and every subsequent visit during treatment
Secondary outcomes:
measure: Severity-weighted Assessment Tool (SWAT) Within up to 12 Months by 2 or More Consecutive Observations Over at Least 4 Weeks
timeFrame: Assessment made at Day 1 and every subsequent visit during treatment
measure: Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)
timeFrame: Baseline to end of therapy
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University Medical Center
state: California
city: Chicago
country: United States
facility: Northwestern University - Dept. of Dermatology
state: Illinois
city: New York
country: United States
facility: NYU Medical Center Dept. of Dermatology
state: New York
city: New York
country: United States
facility: Columbia University, Dept. of Dermatology
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Tulsa
country: United States
facility: Oklahoma University
state: Oklahoma
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Philadelphia
country: United States
facility: Fox Chase Cancer Center
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas, Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: The University of Texas, M.D. Anderson Cancer Center
state: Texas
city: Salt Lake City
country: United States
facility: Utah Clinical Trials, LLC
state: Utah
city: Madison
country: United States
facility: University of Wisconsin
state: Wisconsin
Study officials:
affiliation: Fox Chase Cancer Center
name: Stuart Lessin, M.D.
role: STUDY_DIRECTOR
References:
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.
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.
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.
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.
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.
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.