Pertuzumab Plus Trastuzumab in Patients With Biliary Tract Cancer With ERBB2/3 Alterations: Results From the Targeted Agent and Profiling Utilization Registry Study

Timothy L. Cannon, Michael Rothe, Pam K. Mangat, Elizabeth Garrett-Mayer, Vi K. Chiu, Jimmy Hwang, Namrata Vijayvergia, O. B. Alese, Elie G. Dib, Herbert L. Duvivier, Kelsey A. Klute, Vaibhav Sahai, Eugene R. Ahn, Pablo Bedano, Deepti Behl, Sarah Sinclair, Ramya Thota, Walter J. Urba, Eddy S. Yang, Gina N. GranthamDominique C. Hinshaw, Abigail Gregory, Susan Halabi, Richard L. Schilsky

Research output: Contribution to journalArticlepeer-review

Abstract

PURPOSE: Targeted Agent and Profiling Utilization Registry is a phase II basket trial evaluating the antitumor activity of commercially available targeted agents in patients with advanced cancer and genomic alterations known to be drug targets. Results of a cohort of patients with biliary tract cancer (BTC) with ERBB2/3 amplification, overexpression, or mutation treated with pertuzumab plus trastuzumab are reported. METHODS: Eligible patients had advanced BTC, measurable disease (RECIST v1.1), Eastern Cooperative Oncology Group performance status 0-2, adequate organ function, tumors with ERBB2/3 alterations, and a lack of standard treatment options. Simon's two-stage design was used with a primary end point of disease control (DC), defined as objective response (OR) or stable disease of at least 16+ weeks duration (SD16+) according to RECIST v1.1. Secondary end points included OR, progression-free survival, overall survival, duration of response, duration of stable disease, and safety. RESULTS: Twenty-nine patients were enrolled from February 2017 to January 2022, and all had advanced BTC with an ERBB2/3 alteration. One patient was not evaluable for efficacy. One complete response, eight partial responses, and two SD16+ were observed for DC and OR rates of 40% (90% CI, 27 to 100) and 32% (95% CI, 16 to 52), respectively. The null hypothesis of 15% DC rate was rejected (P = .0015). Four patients had at least one grade 3 adverse event (AE) or serious AE at least possibly related to treatment: anemia, diarrhea, infusion-related reaction, and fatigue. CONCLUSION: Pertuzumab plus trastuzumab met prespecified criteria to declare a signal of activity in patients with BTC and ERBB2/3 amplification, overexpression, or mutation.
Original languageEnglish
Pages (from-to)Jco2302078
JournalJournal of Clinical Oncology
DOIs
StateE-pub ahead of print - May 15 2024

Fingerprint

Dive into the research topics of 'Pertuzumab Plus Trastuzumab in Patients With Biliary Tract Cancer With ERBB2/3 Alterations: Results From the Targeted Agent and Profiling Utilization Registry Study'. Together they form a unique fingerprint.

Cite this