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AzTrials

Non Small Cell Lung CancerPhase 2NCT01813734

Ponatinib in Advanced NSCLC w/ RET Translocations

A Phase 2 study testing Ponatinib in people with Non Small Cell Lung Cancer, run by Massachusetts General Hospital at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Non Small Cell Lung Cancer
What is being tested?
Ponatinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
5 sitesUnited States
Study dates
Sep 2013to Aug 2018
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically or cytologically confirmed advanced NSCLC
  • 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

Who cannot join

  • Clinically significant gastrointestinal abnormalities
  • 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
Show all 15Show fewer
  • 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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • University of Maryland

    Baltimore, Maryland

  • Massachusetts General Hospital

    Boston, Massachusetts, 02115

  • Ohio State University

    Columbus, Ohio, 43210

  • Karmanos Cancer Institute

    Detroit, Michigan

  • University of California, Irvine

    Orange, California, 92868

Other trials in United States

About this study

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

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.

Read the detailed description

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.

Being tested
Ponatinib
Keywords
RET gene mutation
Medical subject headings
Carcinoma, Non-Small-Cell Lung

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PonatinibDrug

    28 day cycle

Study groups in NCT01813734
Study groupRoleWhat they receive
Ponatinib Treatment ArmExperimentalPonatinib 30 mg PO daily
Receives: Drug: Ponatinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate

    The number of participants that achieved either a partial or complete response assessed using Response Evaluation Criteria in Solid Tumors (RECIST v 1.1)

    • Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to < 10 mm.
    • Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.

    Measured: From the start of treatment until disease progression or death, up to approximately 2 years

Secondary outcomes (4)

  1. Disease Control Rate

    The number of participants that achieved either a partial or complete response or stable disease, assessed using Response Evaluation Criteria in Solid Tumors (RECIST v 1.1)

    • Complete Response (CR): Disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to < 10 mm.
    • Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
    • Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease, taking as reference the smallest sum diameters while on study.

    Measured: From the start of treatment until disease progression or death, up to approximately 2 years

  2. Median Progression-Free Survival

    The duration of time from study entry until disease progression (assessed using RECIST 1.1.) or death.

    Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study with at least a 5 mm absolute increase in the sum of all lesions. The appearance of one or more new lesions denotes disease progression.

    Measured: From study entry until disease progression or death, median duration of 3.8 months

  3. 1 Year Overall Survival Rate

    The number of participants surviving one year after study entry

    Measured: 1 year

  4. Number of Participants With Adverse Events

    The number of participants with grade 3 plus adverse events as assessed using Common Toxicology Criteria for Adverse Events (CTCAE 4) that were deemed to be possibly, probably, or definitely related to study treatment.

    Measured: From the start of treatment until 30 days after the end of treatment (up to approximately 2 years)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Mar 2013 (estimated)
  2. Study startSep 2013
  3. Primary completionJun 2018
  4. Study completionAug 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01813734
Sponsor's protocol ID
13-103
Status last verified
Dec 2019
First submitted
13 Mar 2013
Last updated
24 Dec 2019
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 26291023

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ponatinib in people with Non Small Cell Lung Cancer, run by Massachusetts General Hospital at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Dec 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (time frame: From the start of treatment until disease progression or death, up to approximately 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01813734, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Dec 2019.

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