Non-Small Cell Lung Cancer, Head and Neck Cancer Clinical Trial
Official title:
Phase II Study of Ponatinib in Advanced Lung and Head and Neck Cancers With FGFR Kinase Alterations
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.
Status | Terminated |
Enrollment | 3 |
Est. completion date | June 2014 |
Est. primary completion date | June 2014 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years and older |
Eligibility |
Inclusion Criteria: - Measurable disease - Documented evidence of disease progression following most recent therapy - Estimated life expectancy greater than 12 weeks Exclusion Criteria: - 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 |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Beth Israel Deaconess Medical Center | Boston | Massachusetts |
United States | Brigham and Women's Hospital | Boston | Massachusetts |
United States | Dana-Farber Cancer Institute | Boston | Massachusetts |
United States | Massachusetts General Hospital | Boston | Massachusetts |
Lead Sponsor | Collaborator |
---|---|
Dana-Farber Cancer Institute |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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." |
2 years | No |
Secondary | 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 | 2 years | No |
Secondary | 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. | 2 years | No |
Secondary | Define Toxicities of Ponatinib | Number of Participants with Adverse Events as a Measure of Safety and Tolerability | 2 years | Yes |
Secondary | Overall Survival | Measure the overall survival time of patients treated with ponatinib | 2 years | No |
Secondary | Disease Control | Measure the disease control rate of patients treated with ponatinib | 2 years | No |
Secondary | 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. | 2 years | No |
Secondary | 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. | 2 years | No |