Neoplasms

Gland and Hormone Related Diseases

All Conditions

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Thyroid Neoplasms

Thyroid Diseases

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Endocrine Gland Neoplasms

Head and Neck Neoplasms

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Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Adenocarcinoma

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Thyroid Cancer, Medullary

Neuroendocrine Tumor

Neuroepithelioma

Antineoplastic Agents

All Drugs and Chemicals

Amino Acids

Ponatinib

Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Enzyme Inhibitors

Tyrosine

Ponatinib tablets will be taken by mouth, continuously, once daily at a dose of 30 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.

Antonio Fojo, MD

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.

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.

Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer

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

Testing safety and toxicity assessed using CTCAE criteria

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.

PFS is defined as the time from the first day of trial treatment to the first documented disease progression per the Kaplan-Meier curve.

Millennium Pharmaceuticals, Inc.

Antonio Fojo

Assistant Professor of Medicine at the Columbia University Medical

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer	NCT03838692	Medullary Thyro...	Ponatinib	18 Years -	Columbia University
Ponatinib in Advanced or Metastatic Medullary Thyroid Cancer	NCT03838692	Medullary Thyro...	Ponatinib	18 Years -	Columbia University

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