Phase II Study of Ponatinib in Advanced Lung and Head and Neck Cancers With FGFR Kinase Alterations
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 4
- 主要终点
- Response Rate of Patients With Lung or Head and Neck SCC Treated With Ponatinib
研究概览
简要总结
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific cancer. "Investigational" means that the drug is being studied. It also means that the FDA has not yet approved ponatinib for use in patients, including people with your type of cancer.
In order to participate on this study, it must first be determined whether or not a patient's lung or head and neck squamous cell cancer (SCC) has an alteration in FGFR kinase is made from an experimental test on your squamous cell cancer tissue sample. This experimental test is a "genetic test" or "genotyping test", which is a method used to study a tumor's genes. The results are for research purposes only and are not considered "genetic testing" for the purpose of diagnosing medical conditions. Cancers develop as a result of changes that occur in human genetic material (DNA); these changes are called mutations or alterations. This experimental test gives no information about any of the genes in the normal cells of the patient's body, but it helps identify abnormal genes (like FGFR kinase mutations or alterations) usually found only in cancer cells. We will use this experimental test to determine whether or not a tumor contains a required alteration/mutation and thus may respond to ponatinib.
Ponatinib is an investigational, oral anti-cancer drug designed to inhibit abnormal proteins found in cancer cells and may cause those cancer cells to die. In laboratory testing, ponatinib has been shown to inhibit a family of proteins called FGFR kinases, and this genetic alteration/mutation has been found in some squamous cell lung cancers. There is laboratory evidence that alterations/mutations in FGFR kinases in squamous cell lung cancers may be driving the growth of these tumors and that inhibiting these FGFR kinases with ponatinib may decrease or stop the growth of lung SCC.
In this research study, we are looking to see if the study drug, ponatinib, can keep cancer from growing.
详细描述
Patients will be asked to undergo some screening tests or procedures to find out if they can be in the research study. Many of these tests and procedures are likely to be part of regular cancer care and may be done even if it turns out that an individual does not take part in the research study. If a patient has had some of these tests or procedures recently, they may or may not have to be repeated. These screening tests include: medical history, physical examination, vital signs, performance status, assessment of tumor(s), CT scan or MRI scan of brain, routine blood tests, urine pregnancy test for women of childbearing potential and electrocardiogram. Additionally at the time of screening patients will undergo collection of an archival tumor tissue sample for tumor mutation testing.
If a patient takes part in this research study, he or she will be given a study drug-dosing calendar for each treatment cycle. Each treatment cycle lasts 4 weeks during which time the patient will be taking the study drug by mouth daily. The number of cycles the patient will receive will depend on how the patient is tolerating the ponatinib and if your cancer has worsened.
There is a possibility that the following tests or procedures may need to be done at times other than those listed below. These may be done if the research doctors determine they are medically necessary to monitor illness or any side effects a patient may be experiencing. It is important that patients call their research doctor if at any time they are experiencing side effects they cannot tolerate.
During all cycles patients will have a physical exam and be asked questions about their general health and specific questions about any problems that they might be having and any medications they might be taking.
If the patient's disease progresses while you are on this study, they will be given the option of consenting to a new biopsy for research purposes at no financial cost to the patient.
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
- •Measurable disease
- •Documented evidence of disease progression following most recent therapy
- •Estimated life expectancy greater than 12 weeks
排除标准
- •Pregnant or breastfeeding
- •Prior chemotherapy or brain radiotherapy within 4 weeks of entering study
- •Receiving other investigational agents
- •Untreated or progressive brain metastases
- •Prior treatment with or allergic reactions attributed to compounds of similar chemical or biologic composition to ponatinib
- •Known HIV positive on combination antiretroviral therapy
- •Clinically uncontrolled hypertension
- •Previous or concurrent malignancy except adequately treated basal or squamous cell skin cancer, in situ carcinoma of the cervix, or other solid tumor treated curatively and without evidence of recurrence for at least 5 years
- •Active or uncontrolled clinically significant infection
- •Chronic GI disease that may affect bioavailability of ponatinib
- •History of significant bleeding disorder unrelated to cancer
- •Uncontrolled intercurrent illness
- •Clinically significant ventricular arrythmia
- •History of chronic pancreatitis, alcohol abuse or uncontrolled hypertriglyceridemia
研究组 & 干预措施
Ponatinib Treatment Arm
Ponatinib taken by mouth daily
干预措施: ponatinib (Drug)
结局指标
主要结局
Response Rate of Patients With Lung or Head and Neck SCC Treated With Ponatinib
时间窗: 2 years
Investigate the response rate of patients with previously treated lung or head and neck SCC to ponatinib as defined by the proportion of subjects with investigator-assessed confirmed complete response (CR) or partial response (PR). Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR."
次要结局
- Overall Survival(2 years)
- Define the Response Rate to Ponatinib is Patients With FGFR Amplifications Versus Mutations(2 years)
- Prevalence of Specific FGFR Amplifications/Mutations in the Study Population(2 years)
- Progression-free Survival(2 years)
- Define Toxicities of Ponatinib(2 years)
- Disease Control(2 years)
- Determine the Correlation FGFR Amplifications/Mutations With Patient Age, Sex, Disease Stage, Prior Response to Treatment and Smoking History(2 years)
研究者
Peter S. Hammerman
Principal Investigator
Dana-Farber Cancer Institute