Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
9 participants
INTERVENTIONAL
2013-09-30
2018-08-31
Brief Summary
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It has been found that some people with NSCLC have a change (mutation) in a certain gene called the RET gene. This mutated gene may help cancer cells grow. Only participants with a RET mutation will be allowed to participate. In this study, investigators are testing the strategy of using a study drug designed to inhibit or shut off growth signals that results from the mutated RET gene.
Ponatinib is an anti-cancer drug that has been used in research studies for other types of cancer. Ponatinib blocks several growth signals in cancer cells, including RET. In this research study, investigators are looking to see whether ponatinib is effective and safe in treating NSCLC harboring RET rearrangements.
Detailed Description
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Participants will take the study drug once a day, every day of the cycle. Each treatment cycle lasts 28 days (4 weeks). Participants will be given a drug diary to record the drug they take each day. The diary will also include special instructions for taking the study drug.
For cycles 1-20, participants will visit the clinic to have tests and procedures done at the time points listed here: On Day 1-physical exam, vital signs, performance status, electrocardiogram, routine blood tests, pharmacokinetic test, clinical exam and assessment of tumor. On day 15 of cycle 1-vital signs, routine blood tests and clinical exam.
For cycles 11 and on, participants will visit the clinic every 6 weeks. Some of these visits will happen on Day 1 of the cycle and some of these visits will happen on Day 15 of the cycle. At each visit, the following tests and procedures will be done: physical exam, vital signs, performance status, electrocardiogram, routine blood tests, clinical exam, and an assessment of your tumor.
Participants will visit the clinic when their study doctor takes them off study drug permanently. They will also visit the clinic 30 days after stop taking the study drug. The following tests and procedures will be done: physical exam, vital signs, performance status, electrocardiogram, routine blood tests, pregnancy test, clinical exam and a tumor assessment.
Investigators would also like to keep track of participant's medical condition for two years after their first study dose. Investigators will contact participants by telephone every 3 months. If participants still come to the clinic, visits might be used as contact.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Ponatinib Treatment Arm
Ponatinib 30 mg PO daily
Ponatinib
28 day cycle
Interventions
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Ponatinib
28 day cycle
Eligibility Criteria
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Inclusion Criteria
* Molecular confirmation of a RET translocation
* At least one measurable lesion as defined by RECIST
* No restriction on number of prior therapies
* Estimated life expectancy of at least 12 weeks
* Able to swallow and retain orally administered medication
* Must agree to use an effective form of contraception from enrollment through 30 days after the end of study treatment
* Willingness and ability to comply with scheduled visits and study procedures
Exclusion Criteria
* Pregnant or breastfeeding
* Major surgery within 28 days of initiating therapy
* History of CNS disease (Note: Participants with brain metastases will be eligible if treated appropriately and if they remain clinically stable).
* Anti-cancer therapy within 3 weeks
* History of significant bleeding disorder unrelated to cancer
* History of acute pancreatitis within 1 year of study entry or history of chronic pancreatitis
* History of alcohol abuse
* Uncontrolled hypertriglyceridemia
* History of arterial thrombotic events (myocardial infarction, stroke or peripheral vascular disease).
* Uncontrolled hypertension
* Taking medications that are known to be associated with Torsades de Pointes
* Ongoing active infection
* Diagnosed with or received anti-cancer therapy for another primary malignancy within 3 years prior to entry (except for non-melanoma skin cancer or in situ cancers)
* Any condition or illness tha could compromise patient safety or interfere with the evaluation of the drug
18 Years
ALL
No
Sponsors
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Massachusetts General Hospital
OTHER
Responsible Party
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Alice Shaw
Principal Investigator
Locations
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University of California, Irvine
Orange, California, United States
University of Maryland
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Karmanos Cancer Institute
Detroit, Michigan, United States
Ohio State University
Columbus, Ohio, United States
Countries
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References
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Wu H, Shih JY, Yang JC. Rapid Response to Sunitinib in a Patient with Lung Adenocarcinoma Harboring KIF5B-RET Fusion Gene. J Thorac Oncol. 2015 Sep;10(9):e95-e96. doi: 10.1097/JTO.0000000000000611. No abstract available.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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13-103
Identifier Type: -
Identifier Source: org_study_id