NCT01529450已完成不适用
A Pilot Open-Label Study to Examine the Safety and Efficacy of Oral LDE225 in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma Who Have Been Previously Treated With Non-LDE225 Smoothened Inhibitor(s)
Anne Chang1 个研究点 分布在 1 个国家目标入组 11 人开始时间: 2012年2月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 11
- 试验地点
- 1
- 主要终点
- Progression Free Survival (PFS) of All Participants
研究概览
简要总结
This is a prospective single-center, open label, pilot study to investigate the safety and efficacy of LDE225 in patients with locally advanced or metastatic basal cell carcinoma.
Primary Objectives:
• To explore the effects of oral LDE225 on the Progression Free Survival (PFS) of individuals with locally advanced or metastatic BCC who have been previously treated with a non-LDE225 Smo inhibitor.
Secondary Objectives:
- To evaluate the effect of oral LDE225 on tumor tissue biomarkers of BCC activation (Gii 1, 2, Patched 1,2 and Ki67) in individuals which are non-na"ive to Smo inhibitors other than LDE225, at baseline and at end-of-treatment
- To describe adverse effects of oral LDE225 in individuals with a history of non-LDE225 Smo inhibitor usage
- To assess the overall survival rates of individuals with locally advanced BCC or metastatic BCC who have previously taken a non-LDE225 Smo inhibitor after treatment with LDE225
详细描述
This is a prospective single-center, open label, pilot study to investigate the safety and efficacy of LDE225 in patients with locally advanced or metastatic basal cell carcinoma.
Primary Objectives:
• To explore the effects of oral LDE225 on the Progression Free Survival (PFS) of individuals with locally advanced or metastatic BCC who have been previously treated with a non-LDE225 Smo inhibitor.
Secondary Objectives:
- To evaluate the effect of oral LDE225 on tumor tissue biomarkers of BCC activation (e.g. Gli and Ki67) in individuals which are non-naive to Smo inhibitors other than LDE225, at baseline and at end-of-treatment
- To describe adverse effects of oral LDE225 in individuals with a history of non-LDE225 Smo inhibitor usage
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 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
- •Age 18 years or older.
- •Histologically documented diagnosis of basal cell carcinoma deemed to be locally advanced or metastatic who have previously received a non-LDE225 Smo inhibitor.
- •World Health Organization (WHO) performance status <= 2
- •At least one measurable site of disease (as defined by Response Evaluation Criteria in Solid Tumors), or other disease specific response assessment criteria, as appropriate. State age restriction and/or gender/race-ethnic restrictions.
- •Patients with adequate bone marrow, liver and renal function, as specified below:
- •Absolute Neutrophil Count (ANC) >= 1.5 x 10^9/L
- •Hemoglobin (Hgb) >= 9 g/dL
- •Platelets >= 80 x 10^9/L
- •Serum total bilirubin <= 1.5 x upper limit of normal (ULN)
- •Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <= 2.5 x ULN or <= 5 x ULN if liver metastases are present
- •Plasma creatine phosphokinase (CK) < 1.5 x ULN
- •Serum creatinine <= 1.5 x ULN or 24-hour clearance >= 50ml/min
- •Written informed consent obtained prior to any screening procedures
排除标准
- •Patients who have had major surgery within 4 weeks of initiation of study medication.
- •Patients with concurrent uncontrolled medical conditions that may interfere with their participation in the study or potentially affect the interpretation of the study data.
- •State restrictions regarding use of other Investigational Agents.
- •Patients unable to take oral drugs or with lack of physical integrity of the upper gastrointestinal tract or known malabsorption syndromes.
- •State exclusion requirements due to co-morbid disease or incurrent illness, as needed.
- •Patients who have previously been treated with systemic LDE
- •Patients who have neuromuscular disorders (e.g. inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis and spinal muscular atrophy) or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis, such as 3-hydroxy-3-methylglutaryl-coenzyme A (HMG CoA) inhibitors (statins), clofibrate and gemfibrozil, and that cannot be discontinued at least 2 weeks prior to starting LDE225 treatment. If it is essential that the patient stays on a statin to control hyperlipidemia, only pravastatin may be used with extra caution.
- •b) Patients who are planning on embarking on a new strenuous exercise regimen after initiation of study treatment. Note: Muscular activities, such as strenuous exercise, that can result in significant increases in plasma CK levels should be avoided whilst on LDE225 treatment.
- •Patients who have taken part in an experimental drug study within 4 weeks of initiating treatment with LDE
- •Patients who are receiving other anti-neoplastic therapy (e.g. chemotherapy, targeted therapy or radiotherapy) concurrently or within 2 weeks of starting treatment with LDE
- •Patients who are receiving treatment with medications known to be moderate and strong inhibitors or inducers of cytochrome (CYP)3A4/5 or drugs metabolized by CYP2B6 or CYP2C9 that have narrow therapeutic index, and that cannot be discontinued before starting treatment with LDE
- •Medications that are strong CYP3A4/5 inhibitors should be discontinued at least 7 days and strong CYP3A/5 inducers for at least 2 weeks prior to starting treatment with LDE
- •Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotropin (hCG) laboratory test (> 5 mIU/mL).
- •10 Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods. The two methods can be a double barrier method or a barrier method plus a hormonal method. Adequate barrier methods of contraception include:
- •Diaphragm, condom (by the partner), intrauterine device (copper or hormonal), sponge or spermicide. Hormonal contraceptives include any marketed contraceptive agent that includes an estrogen and/or a progestational agent.
- •Reliable contraception should be maintained throughout the study and for 3 months after study drug discontinuation.
- •11 Patients unwilling or unable to comply with the protocol.
研究组 & 干预措施
Refractory Group
Active Comparator
Patients previously treated with non-LDE225 Smo inhibitor who were refractory.
干预措施: LDE225 (Drug)
Resistance Developed Group
Active Comparator
Patients previously treated with non-LDE225 Smo inhibitor who were initially responsive but became resistant with progressive disease.
干预措施: LDE225 (Drug)
结局指标
主要结局
Progression Free Survival (PFS) of All Participants
时间窗: End of treatment or at time of disease progression (up to 58 weeks)
次要结局
- Molecular Markers Associated With Clinical Response(Assessed on day 1)
研究者
Anne Chang
Assistant Professor of Dermatology
Stanford University
研究点 (1)
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