A Phase 2/3 interventional study of ponatinib in Non-Small Cell Lung Cancer, Head and Neck Cancer, sponsored by Dana-Farber Cancer Institute. Terminated at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2014-12-23.
Sponsored by Dana-Farber Cancer Institute · Phase 2/3, Interventional, and Treatment
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.
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.
The investigators would like to keep track of your medical condition for the rest of each patient's life. The investigators would like to do this by calling every six months to see how the patients are doing. Keeping in touch with checking their condition helps the investigators look at the long-term effects of the research study.
2,344 studies on the registry are indexed under Head and Neck Neoplasms; 551 are open to participants now.
This study's enrollment of 3 is below the median of 47 across 1,751 interventional studies indexed under Head and Neck Neoplasms.
Browse Head and Neck Neoplasms studies →Dana-Farber Cancer Institute is the lead sponsor of 813 studies on the registry; 124 are open to participants now.
Of its 113 completed or terminated interventional studies of FDA-regulated products, 77 (68%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Ponatinib taken by mouth daily
Drug: ponatinib
Also known as: AP24534
Response Rate of Patients With Lung or Head and Neck SCC Treated With Ponatinib
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."
Time frame: 2 years
Prevalence of Specific FGFR Amplifications/Mutations in the Study Population
Test tumor DNA using molecular assays to measure the frequency of FGFR amplifications and mutations in study patients
Time frame: 2 years
Progression-free Survival
Establish the progression-free survival of patients with SCC treated with ponatinib as defined by time to development of progression by RECIST criteria.
Time frame: 2 years
Define Toxicities of Ponatinib
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: 2 years
Overall Survival
Measure the overall survival time of patients treated with ponatinib
Time frame: 2 years
Disease Control
Measure the disease control rate of patients treated with ponatinib
Time frame: 2 years
Determine the Correlation FGFR Amplifications/Mutations With Patient Age, Sex, Disease Stage, Prior Response to Treatment and Smoking History
For subjects with FGFR amplifications and for FGFR mutations we will ascertain the age, sex, disease stage, prior response to treatment and smoking history from past medical records and measure whether there are differences in these variables among subjects with amplification versus mutation.
Time frame: 2 years
Define the Response Rate to Ponatinib is Patients With FGFR Amplifications Versus Mutations
Identify the response rate to ponatinib for FGFR specific FGFR amplifications/mutations.
Time frame: 2 years
| Milestone | Ponatinib Treatment Arm |
|---|---|
| Started | 3 |
| Completed | 2 |
| Not completed | 1 |
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."
| percentage of subjects with response | Ponatinib Treatment Arm |
|---|---|
| Response Rate of Patients With Lung or Head and Neck SCC Treated With Ponatinib | 0 |
Test tumor DNA using molecular assays to measure the frequency of FGFR amplifications and mutations in study patients
| participants | Ponatinib Treatment Arm |
|---|---|
| Prevalence of Specific FGFR Amplifications/Mutations in the Study Population | 3 |
Establish the progression-free survival of patients with SCC treated with ponatinib as defined by time to development of progression by RECIST criteria.
| days | Ponatinib Treatment Arm |
|---|---|
| Progression-free Survival | 44 (32 to 55) |
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
| participants | Ponatinib Treatment Arm |
|---|---|
| Define Toxicities of Ponatinib | 2 |
Measure the overall survival time of patients treated with ponatinib
| days | Ponatinib Treatment Arm |
|---|---|
| Overall Survival | 276 (48 to 588) |
Measure the disease control rate of patients treated with ponatinib
| percentage of subjects | Ponatinib Treatment Arm |
|---|---|
| Disease Control | 0 |
For subjects with FGFR amplifications and for FGFR mutations we will ascertain the age, sex, disease stage, prior response to treatment and smoking history from past medical records and measure whether there are differences in these variables among subjects with amplification versus mutation.
No measurements were reported for this outcome.
Identify the response rate to ponatinib for FGFR specific FGFR amplifications/mutations.
No measurements were reported for this outcome.
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ponatinib Treatment Arm | — | 1/2 (50%) | 2/2 (100%) |
| Event | Ponatinib Treatment Arm |
|---|---|
| PancreatitsGastrointestinal disorders | 1/2 |
| Event | Ponatinib Treatment Arm |
|---|---|
| FatigueGeneral disorders | 2/2 |
| HepatitisGastrointestinal disorders | 2/2 |
| DiarrheaGastrointestinal disorders | 1/2 |
| RashSkin and subcutaneous tissue disorders | 1/2 |
| MucositisGastrointestinal disorders | 1/2 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 1/2 |
| MyalgiaMusculoskeletal and connective tissue disorders | 1/2 |
| NeuropathyNervous system disorders | 1/2 |
| Age, Continuous(years) | Ponatinib Treatment Arm |
|---|---|
| Mean | 61 (37 to 74) |
| Sex: Female, Male(Participants) | Ponatinib Treatment Arm |
|---|---|
| Female | 0 |
| Male | 3 |
| Race/Ethnicity, Customized(participants) | Ponatinib Treatment Arm |
|---|---|
| Caucasian | 3 |
| Region of Enrollment(participants) | Ponatinib Treatment Arm |
|---|---|
| United States | 3 |
| Disease(participants) | Ponatinib Treatment Arm |
|---|---|
| Number | 3 |
This study is terminated, as verified in Dec 2014. You cannot join it, but the record below documents what was studied.
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Dana-Farber Cancer Institute