Mycosis FungoidesPhase 2NCT00168064
Safety and Efficacy of Nitrogen Mustard in Treatment of Mycosis Fungoides
A Phase 2 study testing mechlorethamine-MCH (nitrogen mustard) in people with Mycosis Fungoides, run by Yaupon Therapeutics at 12 sites in United States.
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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mycosis Fungoides
- What is being tested?
- mechlorethamine-MCH (nitrogen mustard)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 260 participants
- Where
- 12 sitesUnited States
- Study dates
- May 2006to Aug 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Northwestern University - Dept. of Dermatology
Chicago, Illinois, 61611
University of Texas, Southwestern Medical Center
Dallas, Texas, 75390
Duke University Medical Center
Durham, North Carolina, 27710
The University of Texas, M.D. Anderson Cancer Center
Houston, Texas, 77030
University of Wisconsin
Madison, Wisconsin, 53791
NYU Medical Center Dept. of Dermatology
New York, New York, 10016
Columbia University, Dept. of Dermatology
New York, New York, 10032
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Fox Chase Cancer Center
Philadelphia, Pennsylvania, 19111-2497
Utah Clinical Trials, LLC
Salt Lake City, Utah, 84107
Stanford University Medical Center
Stanford, California, 94305
Oklahoma University
Tulsa, Oklahoma, 74104
About this study
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)
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).
Read the 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.
- Conditions
- Mycosis Fungoides
- Being tested
- mechlorethamine-MCH (nitrogen mustard)
- Keywords
- Mycosis Fungoides, Nitrogen Mustard, Cutaneous T-Cell Lymphoma, CTCL - Mycosis Fungoides
- Medical subject headings
- Mycosis Fungoides, Lymphoma, T-Cell, Cutaneous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mechlorethamine-MCH (nitrogen mustard)Drug
Also called mechlorethamine, 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
| Study group | Role | What they receive |
|---|---|---|
| 1 (PG - NM (MCH) 0.02%) | Active comparator | 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 Receives: Drug: mechlorethamine-MCH (nitrogen mustard) |
| 2 (AP - MCH(NM) 0.02%) | Active comparator | 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 Receives: Drug: mechlorethamine-MCH (nitrogen mustard) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Assessment made at Day 1 and every subsequent visit during treatment
Secondary outcomes (2)
Severity-weighted Assessment Tool (SWAT) Within up to 12 Months by 2 or More Consecutive Observations Over at Least 4 Weeks
Measured: 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)
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.
Measured: Baseline to end of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2005 (estimated)
- Study startMay 2006
- Primary completionAug 2010
- Study completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Mycosis FungoidesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 260 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yaupon Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stuart Lessin, M.D., Study director, Fox Chase Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00168064
- Sponsor's protocol ID
- 2005NMMF-201-US
- Status last verified
- Oct 2012
- First submitted
- 7 Sep 2005
- Last updated
- 31 Oct 2012
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
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.
PubMed 12588222Kim 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.
PubMed 14686971Kim 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.
PubMed 8915308Geskin LJ, Angello JT, Bagot M, Guenova E, Nikbakht N, Querfeld C, Scarisbrick JJ. Evaluating Response Trends of Chlormethine/Mechlorethamine Gel in Patients With Stage I-IIA Mycosis Fungoides: Analysis of Individual Patient Data From a Randomized Controlled Phase II Study to Facilitate Optimal Treatment Experiences. Clin Lymphoma Myeloma Leuk. 2024 Jan;24(1):40-47. doi: 10.1016/j.clml.2023.08.020. Epub 2023 Sep 7.
PubMed 37802679Querfeld 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.
PubMed 35536441Querfeld 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.
PubMed 34091453Lessin 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.
PubMed 23069814
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT00168064
- Acronym / sponsor ID
- 2005NMMF-201-US
- Conditions
- Mycosis Fungoides
- Keywords
- Mycosis Fungoides, Nitrogen Mustard, Cutaneous T-Cell Lymphoma, CTCL - Mycosis Fungoides
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 260 participants (actual)
- Lead sponsor
- Yaupon TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- May 2006
- Primary completion
- Aug 2010 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 14 Sep 2005 (estimated)
- Results
- Posted 31 Oct 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 (PG - NM (MCH) 0.02%) | Active comparator | Drug: 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%) | Active comparator | Drug: 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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mechlorethamine-MCH (nitrogen mustard)Also: mechlorethamine, nitrogen mustardAll 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 | Drug | 1 (PG - NM (MCH) 0.02%), 2 (AP - MCH(NM) 0.02%) |
Primary outcome
Ratio of Response Rates Based on CAILS
Time frame: 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
2 secondary outcomes
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
Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)
Time frame: Baseline to end of therapy
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 12588222Kim 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.
- Background: PubMed 14686971Kim 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.
- Background: PubMed 8915308Kim 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.
- Derived from this study: PubMed 37802679Geskin LJ, Angello JT, Bagot M, Guenova E, Nikbakht N, Querfeld C, Scarisbrick JJ. Evaluating Response Trends of Chlormethine/Mechlorethamine Gel in Patients With Stage I-IIA Mycosis Fungoides: Analysis of Individual Patient Data From a Randomized Controlled Phase II Study to Facilitate Optimal Treatment Experiences. Clin Lymphoma Myeloma Leuk. 2024 Jan;24(1):40-47. doi: 10.1016/j.clml.2023.08.020. Epub 2023 Sep 7.
- Derived from this study: PubMed 35536441Querfeld 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.
- Derived from this study: PubMed 34091453Querfeld 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.
- Derived from this study: PubMed 23069814Lessin 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Stuart Lessin, M.D. study director
Fox Chase Cancer Center
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing mechlorethamine-MCH (nitrogen mustard) in people with Mycosis Fungoides, run by Yaupon Therapeutics at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Oct 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ratio of Response Rates Based on CAILS (time frame: Assessment made at Day 1 and every subsequent visit during treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00168064, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Oct 2012.