Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module - 5 LDE225 for Patients With PTCH1 or SMO Mutated Tumors
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 10
- 试验地点
- 10
- 主要终点
- Summary of Overall Response (ORR) and Clinical Benefit (CBR)
研究概览
简要总结
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
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
- •Patient has confirmed diagnosis of a select solid tumor (except medulloblastoma, basal cell carcinoma and pancreatic adenocarcinoma) or hematological malignancy (except CML, ALL and AML).
- •Patient has pre-identified tumor with a PTCH1 or SMO mutation.
- •Patient has received at least one prior treatment for recurrent, metastatic and /or locally advanced disease and for whom no standard therapy options are anticipated to result in a durable remission.
- •Patient has progressive and measurable disease as per RECIST 1.
- •or other appropriate hematological guidelines.
- •Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
排除标准
- •Patients has received prior treatment with LDE
- •Patients has neuromuscular disorders associated with elevated CK (i.e. inflammatory myopathies, muscular dystrophy, amyotrophic lateral sclerosis and spinal muscular atrophy) or are on concomitant treatment with drugs that are recognized to cause rhabdomyolysis
- •Patients has primary CNS tumor or CNS tumor involvement
- •Patient has received chemotherapy or anticancer therapy ≤ 4 weeks prior to starting study drug
研究组 & 干预措施
LDE225
LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule.
干预措施: LDE225 (Drug)
结局指标
主要结局
Summary of Overall Response (ORR) and Clinical Benefit (CBR)
时间窗: 16 weeks
Clinical benefit rate (CBR) Number and percentage of subjects with CBR (responses of CR, PR or SD ≥ 16 weeks) as assessed by investigator was reported for all patients along with 95% exact confidence interval (CI). Overall Response Rate (ORR) Overall response was to be determined by investigator assessment for each tumor in the study. For subjects with solid tumors, the assessment criteria was RECIST 1.1 and included responses of CR and/or PR. The number and percentage of subjects for different categories of overall response (e.g., for solid tumors - CR, PR, SD, PD, Not Evaluable) were to be provided for solid tumors, and each hematological tumor type (if applicable). Ninety-five percent (95%) exact CI was to be provided for the response rate(s) (e.g., for solid tumors - CRn and/or PR) as well.
次要结局
- Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set(4 months)
- Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months(4 months)