NCT02265341

Ponatinib Hydrochloride in Treating Patients With Advanced Biliary Cancer With FGFR2 Fusions

Pilot Study of Ponatinib in Biliary Cancer Patients With FGFR2 Fusions

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This pilot phase II trial studies how well ponatinib hydrochloride works in treating patients with biliary cancer that has spread to other places in the body and that have alterations (fusions) in a gene known as fibroblast growth factor receptor 2 (FGFR2). Ponatinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVES: I. To assess the clinical benefit rate (confirmed complete or partial response or stable disease for 4 or more cycles) of ponatinib (ponatinib hydrochloride) in fibroblast growth factor receptor (FGFR) aberrant advanced biliary cancers. SECONDARY OBJECTIVES: I. To estimate progression free survival, overall survival, and cancer antigen 19-9 (CA19-9) response rate of these patients. II. To estimate the adverse event profile of ponatinib. TERTIARY OBJECTIVES: I. Establish preliminary correlations between FGFR2 fusions and evidence of any clinical benefit. II. Assess preliminary evaluation of FGFR2 pathway perturbation with ponatinib. III. To describe patient-reported health-related quality of life and symptoms. 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 every 6 months for at least 2 years.

Conditions

• Malignant Hepatobiliary Neoplasm

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Ponatinib Hydrochloride	Given PO
OTHER	Quality-of-Life Assessment	Ancillary studies

Timeline

• Start date: 2014-12-01
• Primary completion: 2018-05-01
• Completion: 2019-06-14
• First posted: 2014-10-15
• Last updated: 2020-11-25
• Results posted: 2019-08-06

Locations

1 site across 1 country: United States