NCT02272998

Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT.

Phase II Study of Ponatinib for Advanced Cancers With Genomic Alterations in Fibroblastic Growth Factor Receptor (FGFR) and Other Genomic Targets (KIT, PDGFRá, RET FLT3, ABL1)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Sameek Roychowdhury · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

PRIMARY OBJECTIVES: I. To evaluate the response of ponatinib (ponatinib hydrochloride) in patients with fibroblast growth factor receptor (FGFR) altered cancers. SECONDARY OBJECTIVES: I. To assess the safety and tolerability of ponatinib in advanced solid tumors with genomic FGFR alterations. 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). OUTLINE: 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.

Conditions

• Malignant Neoplasm

Interventions

Type	Name	Description
DRUG	ponatinib hydrochloride	Given PO
OTHER	laboratory biomarker analysis	Correlative studies

Timeline

• Start date: 2015-02-24
• Primary completion: 2022-11-17
• Completion: 2022-11-17
• First posted: 2014-10-23
• Last updated: 2025-03-24
• Results posted: 2025-03-24

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study