A Randomized Placebo-Controlled Phase II Trial Evaluating the Effect of Hedgehog Inhibitor LDE225 on Bone Marrow Disseminated Tumor Cells in Women With Early Stage Estrogen Receptor Negative and HER2 Negative Breast Cancer
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Proportion of patients who are bone marrow DTC-negative after therapy
研究概览
简要总结
This randomized phase II trial studies the effects of erismodegib (LDE225) on disseminated tumor cells (DTCs) in patients with stage III-III estrogen receptor (ER)-negative and human epidermal growth factor receptor 2 (HER2)-negative breast cancer. The presence of DTCs after completing treatment for breast cancer may be linked to recurrence of the disease. LDE225 may eliminate DTCs in bone marrow and reduce the risk of recurrence.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Arm I (erismodegib [LDE225])
400 mg daily and treatment repeats every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity.
干预措施: Erismodegib (Drug)
Arm II (placebo)
Patients receive placebo PO daily and treatment repeats every 28 days for up to 26 cycles in the absence of disease progression or unacceptable toxicity.
干预措施: Placebo (Drug)
结局指标
主要结局
Proportion of patients who are bone marrow DTC-negative after therapy
时间窗: at 6 months
Comparing LDE225 to placebo using a stratified Cochran-Mantel-Haenszel test for difference of proportions
次要结局
- Disease-free survival (DFS)(2 years from initiation of study treatment)
- Overall Survival (OS)(2 years from initiation of study treatment)
- Ptch1 expression after treatment with LDE225 or placebo(At 6 months)
- Incidence of toxicities associated with LDE225 or placebo treatment(For 30 days following the last day of study treatment; up to 25 months)
- Time to recurrence and death in DTC-negative patients and DTC-positive patients(2 years from initiation of study treatment)
- Time to recurrence and death in ICC negative versus ICC positive patients(2 years from initiation of treatment)
- Concordance of DTC determination by ICC or gene expression(Baseline)