This phase II trial studies how well ponatinib hydrochloride works in treating patients with
cancer that has spread to other parts of the body (metastatic), has failed previous treatment
(refractory), and has one of several alterations, or mutations, in its deoxyribonucleic acid
(DNA) sequence. Ponatinib hydrochloride may stop the growth of cancer cells by blocking some
of the enzymes needed for cell growth. It is not yet known whether a patient's genetic
alterations may affect how well ponatinib hydrochloride works.
I. To evaluate the response of ponatinib (ponatinib hydrochloride) in patients with
fibroblast growth factor receptor (FGFR) altered cancers.
I. To assess the safety and tolerability of ponatinib in advanced solid tumors with genomic
II. To assess progression free survival (PFS) and overall survival (OS) with ponatinib.
III. To determine candidate genomic and proteomic biomarkers of sensitivity and resistance to
ponatinib using unbiased high throughput approaches (exome, transcriptome, reverse phase
protein array [RPPA]).
IV. To assess response of ponatinib in advanced cancers with subsets of genomic FGFR
alterations (fusions vs. amplifications vs. mutations).
Patients receive ponatinib hydrochloride orally (PO) once daily (QD) on days 1-28. Courses
repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 52 weeks.
Ewing's Family Tumors,
primary malignant neoplasm,
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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