NCT03838692

Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer

A Study of Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer (MTC)

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Antonio Fojo · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to determine in an exploratory manner the objective overall response rate to ponatinib in the treatment of patients with advanced or metastatic MTC previously treated with cabozantinib or vandetanib who have tumors with rearranged-during-transfection (RET) mutations and have tumors without RET mutations.

Detailed description

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: 1. 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. 2. Determine the side effects that may occur with this treatment. 3. 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.

Conditions

• Medullary Thyroid Cancer

Interventions

Type	Name	Description
DRUG	Ponatinib	A daily oral dose at 30 mg.

Timeline

• Start date: 2019-07-26
• Primary completion: 2021-06-14
• Completion: 2021-06-14
• First posted: 2019-02-12
• Last updated: 2021-10-13

Regulatory

• FDA-regulated drug study