Ponatinib in Advanced NSCLC w/ RET Translocations
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01813734|
Recruitment Status : Completed
First Posted : March 19, 2013
Last Update Posted : November 9, 2018
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 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.
|Condition or disease||Intervention/treatment||Phase|
|Non Small Cell Lung Cancer||Drug: Ponatinib||Phase 2|
Participants in this research study will be asked to undergo some screening tests or procedures to confirm that eligibility. 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 participants do not take part in the research study. These tests and procedures include the following: medical history, vital signs, physical exam, performance status, electrocardiogram, echocardiogram, routine blood tests, pregnancy test and an assessment of tumor by CT or MRI. If these tests show that a participant is eligible to participate in the research study, he/she will begin the study treatment. If a patient does not meet the eligibility criteria, he/she will not be able to participate in this research study.
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II, Open-Label Study of Ponatinib, A Multi-Targeted Oral Tyrosine Kinase Inhibitor, in Advanced Non-Small Cell Lung Cancer Harboring RET Translocations|
|Study Start Date :||September 2013|
|Actual Primary Completion Date :||June 2018|
|Actual Study Completion Date :||August 2018|
Experimental: Ponatinib Treatment Arm
Ponatinib 30 mg PO daily
28 day cycle
- Overall Response Rate [ Time Frame: 2 years ]To evaluate the overall response rate (ORR) of ponatinib in RET-positive NSCLC as assessed by RECIST criteria
- Disease Control Rate [ Time Frame: 2 years ]
- Progression-Free Survival [ Time Frame: 2 years ]
- 1 Year Overall Survival Rate [ Time Frame: 1 year ]
- Number of participants with adverse events [ Time Frame: 2 years ]To determine the safety and tolerability of ponatinib in patients with RET translocation positive NSCLC
- Plasma concentrations of ponatinib [ Time Frame: 2 years ]To determine the plasma pharmacokinetics of ponatinib
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01813734
|United States, California|
|University of California, Irvine|
|Orange, California, United States, 92868|
|United States, Maryland|
|University of Maryland|
|Baltimore, Maryland, United States|
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02115|
|United States, Michigan|
|Karmanos Cancer Institute|
|Detroit, Michigan, United States|
|United States, Ohio|
|Ohio State University|
|Columbus, Ohio, United States, 43210|