Modular Phase II Study to Link Targeted Therapy to Patients With Pathway Activated Tumors: Module 1 - BKM120 for Patients With PI3K-activated Tumors
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 146
- 试验地点
- 59
- 主要终点
- Participant Clinical Benefit Response Rate
研究概览
简要总结
The purpose of this signal seeking study was is to determine whether treatment with BKM120 demonstrates sufficient efficacy in select pathway-activated 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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient had a confirmed diagnosis of a solid tumor or hematological malignancy with the exception of endometrial cancer, glioblastoma, nonsmall cell lung cancer, prostate cancer or breast cancer.
- •Patient's tumor was evaluated and pre-identified to have activation of the PI3K pathway, at a CLIA certified laboratory
- •Patient must have received at least one prior treatment for recurrent metastatic and /or locally advanced disease and for whom no standard therapy options was anticipated to result in a durable remission.
- •Patient must have had progressive and measurable disease as per RECIST 1.
- •or other appropriate hematological guidelines
- •Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
排除标准
- •Patient had received previous treatment with BKM120 Patient had symptomatic CNS metastases Patient had mood disorder as outlined in Section 5 Patient had received chemotherapy or other anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.
研究组 & 干预措施
BKM120
BKM120 100 mg (oral gelatine capsules) was administered orally once daily starting from cycle 1 day 1 and will be dosed continuously every day for each 28- day cycle
干预措施: BKM120 (Drug)
结局指标
主要结局
Participant Clinical Benefit Response Rate
时间窗: Week 16
Clinical benefit rate for patients with solid tumors will be assessed using RECIST 1.1 and will include responses of Complete Response (CR) or Partial Response (PR) or Stable Disease (SD) at \>=16 weeks. For hematologic tumors other appropriate hematological response criteria was applied. Response criteria: CR=Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm., PR=At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters, SD=Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study, PD= At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline
次要结局
- Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Rate in Percentages(baseline up to 24 months)
- Overall Survival - Number of Participants With an Event(Every 8 Weeks until death, assessed up to 24 months)
- Overall Survival (OS)- Kaplan-Meier Estimates of OS Timing in Months(baseline up to 24 months)
- Overall Response of Partial Response (PR) or Greater. PR=at Least a 30% Decrease in the Sum of Diameters of Target Lesions, Taking as Reference the Baseline Sum Diameters(baseline and every 8 weeks until disease progression or end of treatment, assessed up to 24 months)
- Progression-Free Survival - Number of Participants With an Event(Every 8 Weeks until death, assessed up to 24 months)
- Progression-Free Survival (PFS)- Kaplan-Meier Estimates of PFS Timing in Months(baseline up to 24 months)
- Overall Survival (OS)- Kaplan-Meier Estimates of OS Rate in Percentages(baseline up to 30 months)