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)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 260
- 试验地点
- 12
- 主要终点
- Ratio of Response Rates Based on CAILS
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
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
干预措施: mechlorethamine-MCH (nitrogen mustard) (Drug)
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
干预措施: mechlorethamine-MCH (nitrogen mustard) (Drug)
结局指标
主要结局
Ratio of Response Rates Based on CAILS
时间窗: 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
次要结局
- 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)
- Percent of Participants Achieving at Least 50% Improvement of Severity Weighted Assessment Tool (SWAT)(Baseline to end of therapy)