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Clinical Trial
. 2024 Oct:8:e2400477.
doi: 10.1200/PO-24-00477. Epub 2024 Oct 16.

Palbociclib in Patients With Head and Neck Cancer and Other Tumors With CDKN2A Alterations: Results From the Targeted Agent and Profiling Utilization Registry Study

Francis P Worden  1 , Evan Pisick  2 , Michael Rothe  3 , Pam K Mangat  3 , Elizabeth Garrett-Mayer  3 , Maged F Khalil  4 , Daniel R Carrizosa  5 , Jessica R Bauman  6 , Rom S Leidner  7 , Herbert L Duvivier  8 , Siqing Fu  9 , Min S Park  10 , Kathleen J Yost  11 , Carmen J Calfa  12 , Alissa S Marr  13 , Ani S Balmanoukian  14 , Deepti Behl  15 , Timothy L Cannon  16 , Lisle Nabell  17 , Steven Francis Powell  18 , Ramya Thota  19 , Dominique C Hinshaw  3 , Abigail Gregory  3 , Gina N Grantham  3 , Susan Halabi  20 , Richard L Schilsky  3
Affiliations
Clinical Trial

Palbociclib in Patients With Head and Neck Cancer and Other Tumors With CDKN2A Alterations: Results From the Targeted Agent and Profiling Utilization Registry Study

Francis P Worden et al. JCO Precis Oncol. 2024 Oct.

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 targetable genomic alterations. Two cohorts of patients with cyclin-dependent kinase inhibitor 2A (CDKN2A)-mutated tumors treated with palbociclib are reported: one with head and neck cancer (HNC) with both squamous and nonsquamous cell histologies, and one with histology-pooled (HP) cancers.

Methods: Eligible patients had measurable disease, Eastern Cooperative Oncology Group performance status 0-2, adequate organ function, and no standard treatment options. The primary end point was disease control (DC), defined as objective response (OR) or stable disease (SD) of at least 16+ weeks duration. For the HNC cohort, Simon's two-stage design with a null DC rate of 15% versus 35% (power = 0.85; α = .10) was used. For the HP cohort, the null hypothesis of a DC rate of 15% was rejected if the lower limit of a one-sided 90% CI was >15%. Secondary end points included OR, safety, progression-free survival, overall survival, duration of response, and duration of SD.

Results: Seventy patients with HNC (N = 28) or HP cancers (N = 42) were treated with palbociclib. For the HNC cohort, DC and OR rates were 40% (one-sided 90% CI, 27 to 100) and 4% (95% CI, <1 to 18), respectively. The null hypothesis was rejected (P = .002). For the HP cohort, DC and OR rates were 13% (one-sided 90% CI, 6 to 100) and 5% (95% CI, <1 to 17), respectively. The null hypothesis was not rejected. Thirty-one of 70 patients experienced treatment-related grade 3 to 4 adverse events (AEs) or serious AEs, the most common including neutropenia, thrombocytopenia, and leukopenia.

Conclusion: Palbociclib met prespecified criteria to declare a signal of activity in patients with HNC with CDKN2A alterations, but not in the HP cohort.

Trial registration: ClinicalTrials.gov NCT02693535.

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