Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer
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|ClinicalTrials.gov Identifier: NCT03838692|
Recruitment Status : Not yet recruiting
First Posted : February 12, 2019
Last Update Posted : February 12, 2019
|Condition or disease||Intervention/treatment||Phase|
|Medullary Thyroid Cancer||Drug: Ponatinib||Phase 2|
Ponatinib is an investigational agent that blocks abnormal cancer proteins and therefore harms cancer cells. It was recently approved by the Food and Drug Administration for leukemia treatment, but is not approved for medullary thyroid cancer treatment. Early studies, however, have shown that medullary thyroid tumors respond to ponatinib. These studies were done in the laboratory and not performed on humans. This is a study designed for patients with medullary thyroid cancer. Treatment will consist of a drug called ponatinib which is a pill that taken once each day and continue throughout the duration of the study. The purpose of the study is to:
- Determine if this treatment will be effective in decreasing the amount of cancer and, if it does, to determine how long the response will last.
- Determine the side effects that may occur with this treatment.
- To analyze genetic mutations in tumors to help us understand how tumors grow and how these drugs interact with the mechanisms within the tumor.
Ponatinib has been given to many patients with leukemia and is now approved by the United States Food and Drug Administration for treatment of certain types of leukemia. This study will seek to determine if ponatinib can bring benefit to patients with medullary thyroid cancer.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||31 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Study of Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer (MTC)|
|Estimated Study Start Date :||April 2019|
|Estimated Primary Completion Date :||December 2020|
|Estimated Study Completion Date :||June 2021|
Experimental: Ponatinib Arm
Ponatinib tablets will be taken by mouth, continuously, once daily at a dose of 45 mg. A cycle of ponatinib is defined as 28 consecutive days starting with the first day of the treatment cycle. Treatment can be taken with water, with or without food, at approximately the same time each day.
A daily oral dose at 45 mg.
Other Name: ICLUSIG
- Objective Response Rate (ORR) [ Time Frame: 24 months ]ORR is defined as the proportion of the subjects in the analysis population who have a complete response (CR) or partial response (PR). Responses are based on assessments per RECIST.
- Progression-Free Survival (PFS) [ Time Frame: 24 months ]PFS is defined as the time from the first day of trial treatment to the first documented disease progression per the Kaplan-Meier curve.
- Total Number of Adverse Reactions [ Time Frame: 24 months ]Testing safety and toxicity assessed using CTCAE criteria
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): NCT03838692
|Contact: Dan Otapfirstname.lastname@example.org|
|Contact: Antonio Fojo, MD.||212 305 email@example.com|
|Principal Investigator:||Antonio Fojo, MD||Columbia University|