Therapeutic Efficacy of Topical Sirolimus in Early Stage Cutaneous T-cell Lymphoma (CTCL)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- Overall Response Rate
研究概览
简要总结
The purpose of this study is to determine whether sirolimus reduces the symptoms of cutaneous T-cell lymphoma (CTCL) and whether it causes any side effects.
详细描述
This will be a prospective, non-randomized, open label study of topical sirolimus for the treatment of CTCL recurrent or refractory to at least one previous skin directed treatment. The purpose will be evaluation of safety and anti-tumor response as evaluated by serial skin examinations.
Study duration:
For subjects with at least partial remission, treatment will be continued for a maximum of 6 months. All subjects will be followed for 6 months from the time of discontinuation of the study drug or until progression of disease or until a new treatment for CTCL will be started.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinically and histologically confirmed diagnosis of CTCL (early stage disease with patches and/or thin plaques covering up to 10%, stage IA)
- •Relapsed or refractory disease after at least one standard skin directed treatment including corticosteroids, topical bexarotene, phototherapy
- •All subjects must be 18 years of age or older
- •Life expectancy ≥ 6 months, determined by the treating physician
- •Signed informed consent
排除标准
- •Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of the start of study drug (including topical or systemic glucocorticosteroids, chemotherapy, radiation therapy, antibody based therapy, etc.)
- •Prior treatment with any investigational drug within the preceding 4 weeks
- •Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent.
- •Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. Adequate contraception (oral contraceptives ("the pill"), intrauterine devices (IUDs), contraceptive implants under the skin, or contraceptive injections, diaphragms with spermicide and condoms with foam) must be used throughout the trial and for 8 weeks after the last dose of study drug (women of childbearing potential must have a negative urine within 7 days prior to administration of sirolimus).
- •Patients who have received prior treatment with an mTOR inhibitor (e.g., sirolimus, temsirolimus, everolimus).
- •Patients with a known hypersensitivity to sirolimus or other rapamycin (e.g., everolimus, temsirolimus) or to its excipient
- •History of noncompliance to medical regimens
- •Patients unwilling to or unable to comply with the protocol
研究组 & 干预措施
Sirolimus 0.1% ointment
Sirolimus 0.1% ointment
干预措施: Sirolimus 0.1% Ointment (Drug)
结局指标
主要结局
Overall Response Rate
时间窗: 6 months
Determine the efficacy of topical sirolimus in the treatment of early stage CTCL as overall response rate (ORR) Response criteria: Complete response (CR) will be defined as no evidence of clinical skin disease. Partial response (PR) will be defined as a marked improvement in skin disease of \> 50% from baseline without new lesions. Stable disease (SD) will be defined as \< 25% increase and \< 50% clearance in skin disease from baseline without new lesions. Progressive disease (PD) will be defined as \> 25% increase in skin disease from baseline or new tumors or loss of response in those with CR or PR (increase in skin score of greater than the sum of nadir plus 50% baseline score). The assessment will be based on Composite Assessment of Index Lesion Severity (CAILS) and, in case of more extensive disease, Modified Severity-Weighed Assessment Tool (mSWAT) scores.
次要结局
- Duration of objective response (DOR)(6 months)
- Adverse event profile and tolerability of topical sirolimus in patients with CTCL(6 months)
- Correlative biomarkers such as mTOR pathway activation at baseline and during treatment(6 months)
研究者
Stefan Schieke MD
Assistant Professor
Medical College of Wisconsin