Ponatinib for Squamous Cell Lung and Head and Neck Cancers

NCT01761747 | Terminated Phase 2 Results DMC

• 1 other identifier: 12-327
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Non-Small Cell Lung Cancer, Head and Neck Cancer

Keywords

Squamous Cell Carcinoma

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (7)

• Prevalence of Specific FGFR Amplifications/Mutations in the Study Population

  2 years

• Progression-free Survival

  2 years

• Define Toxicities of Ponatinib

  2 years

• Overall Survival

  2 years

• Disease Control

  2 years

Study Arms (1)

Ponatinib Treatment Arm

EXPERIMENTAL

Ponatinib taken by mouth daily

Drug: ponatinib

Interventions

ponatinib DRUG

Also known as: AP24534

Ponatinib Treatment Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Measurable disease
• Documented evidence of disease progression following most recent therapy
• Estimated life expectancy greater than 12 weeks

You may not qualify if:

• 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

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead

Study Sites (4)

Massachusetts General Hospital

Boston, Massachusetts, 02214, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02215, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

MeSH Terms

Conditions

Carcinoma, Non-Small-Cell Lung; Head and Neck Neoplasms; Carcinoma, Squamous Cell

Interventions

ponatinib

Condition Hierarchy (Ancestors)

Carcinoma, Bronchogenic; Bronchial Neoplasms; Lung Neoplasms; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Lung Diseases; Respiratory Tract Diseases; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms, Squamous Cell

Results Point of Contact

• Title: Dr. Peter Hammerman
• Organization: Dana-Farber Cancer Institute

phammerman@partners.org

617-632-6335

Study Officials

• Peter Hammerman, MD, PhD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 26, 2012
• First Posted: January 7, 2013
• Study Start: January 1, 2013
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2014
• Last Updated: December 23, 2014
• Results First Posted: December 23, 2014

Record last verified: 2014-12

Locations

US (4)