A Pilot Clinical Trial of Genomic Based Assignment of Therapy in Advanced Urothelial Carcinoma
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Percentage of Participants Who Enrolled and Underwent a Biopsy Who Went on to Receive Treatment Within 21 Days
研究概览
简要总结
Background:
Advanced urothelial cancer has no cure. But only a few chemotherapy drugs have been tested for it. The Co-eXpression ExtrapolatioN (COXEN) model predicts if cells respond to treatment. It may also help determine which drugs fight urothelial cancer based on the characteristics of a tumor. Researchers want to test if this model can choose the best therapy for advanced urothelial cancer within 3 weeks and how tumors respond to the next best therapy.
Objective:
To test if the COXEN model can choose the best therapy for advanced urothelial cancer within 3 weeks.
Eligibility:
People ages 18 and older whose urothelial cancer has spread after at least 1 line of chemotherapy
Design:
Participants will be screened with medical history, physical exam, blood and urine tests, and tumor scans.
Participants will provide a tumor sample from a previous surgery and a new biopsy. A needle will remove a small piece of tumor.
Participants will repeat screening tests, plus have an electrocardiogram (EKG) and scan. For the scan, they will get an injection of radioactive drug. They will lie in a machine that takes pictures.
Participants will take the drugs assigned by the COXEN model. They will have visits every 2-3 weeks. These will include blood and urine tests.
Participants will have tumor scans every 8-9 weeks.
Participants may have another biopsy.
Participants will take the drugs until they can't tolerate the side effects or their cancer worsens. They may be assigned to a second COXEN therapy.
Participants will have a follow-up visit 4-5 weeks after their last drug dose.
Participants will be contacted by phone every few months until death.
详细描述
Background:
- Patients presenting de novo with metastatic bladder cancer, or developing visceral metastatic disease after local treatment, are incurable with currently available therapeutic modalities.
- Only a small number of chemotherapeutic agents have been tested and very few have some single agent activity in the treatment of metastatic urothelial carcinoma. However most (>100) Food and Drug Administration (FDA) approved anticancer agents have yet to be tested in this disease.
- Novel approaches to the development of genomic predictors of chemosensitivity that do not require clinical trials for their identification are urgently needed in order to identify agents that are clinically effective when either repurposed or discovered de novo specifically for urothelial carcinoma. Such repurposing of an FDA approved anticancer agent in order to advance therapy from one cancer to another would require only minimal clinical development, saving billions of dollars and reducing the time required to reach routine clinical practice.
- Our established extramural-intramural National Cancer Institute (NCI) collaboration pulls together significant expertise in biomarker development and clinical trial design in bladder cancer. The innovation of this group lies not only in the novel scientific approaches i.e. CoeXpression ExtrapolatioN (COXEN) under investigation, but also in the successful creation of a cohesive multi-institutional research collaboration dedicated to improved clinical outcomes in bladder cancer patients.
- COXEN uses molecular profiles as a Rosetta Stone for translating drug sensitivities of one set of cancers into predictions for another completely independent set of cell lines or human tumors. The COXEN methodology has been scrutinized and deemed methodologically sound by peer review. The ability of COXEN to predict drug effectiveness in patients a priori, from purely in vitro assays, is unique as no other tool currently either in practice or in development provides similar results.
Objectives:
- To determine the feasibility of using the Co-eXpression ExtrapolatioN (COXEN) model in making a real-time treatment decision (within 3 weeks) in patients with advanced urothelial carcinoma.
Eligibility:
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Treatment Regimen
Treatment regimen selected by CO eXpression ExtrapolatioN (COXEN) model
干预措施: 75 approved agents (Drug)
Treatment Regimen
Treatment regimen selected by CO eXpression ExtrapolatioN (COXEN) model
干预措施: COXEN (Other)
结局指标
主要结局
Percentage of Participants Who Enrolled and Underwent a Biopsy Who Went on to Receive Treatment Within 21 Days
时间窗: time to treatment assignment, approximately 3 weeks
Participants were assigned a treatment combination by the co-expression extrapolation (COXEN) algorithm. The COXEN algorithm used a multi-step process that involved pathology, tissue processing, messenger ribonucleic acid (mRNA) profiling and bioinformatics, etc. to select a treatment regimen.
次要结局
- Progression Free Survival(Every 2 cycles until progression, approximately 4 months.)
- Overall Survival(From date of treatment content until the date of death from any cause or date off study, whichever came first, assessed up to 10 months and 16 days.)
- Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0)(Date treatment consent signed to date off study, approx. 5 mos/ 6 dys for the 1st Grp; 10 mos/16 dys for the 2nd Grp; 8 mos/13 dys for the 3rd Grp; 5 mos/16 dys for the 4th Grp; and 27 dys for the 5th Grp.)
- Proportion of Patients With an Objective Response(End of treatment, approximately 4 months.)
- Number of Participants Who Had Adverse Events ≥ Grade 1(Date treatment consent signed to date off study, approx. 5 mos/ 6 dys for the 1st Grp; 10 mos/16 dys for the 2nd Grp; 8 mos/13 dys for the 3rd Grp; 5 mos/16 dys for the 4th Grp; and 27 dys for the 5th Grp.)
研究者
Andrea Apolo, M.D.
Principal Investigator
National Cancer Institute (NCI)