Pembrolizumab in advanced osteosarcoma: results of a single-arm, open-label, phase 2 trial.

ABSTRACT:

Aim

To evaluate the activity and safety of the PD-1 antibody pembrolizumab in adult patients with advanced osteosarcoma.

Material and methods

The study was a single-arm, open-label, phase 2 trial in patients with unresectable, relapsed osteosarcoma. The primary endpoint was clinical benefit rate (CBR) at 18 weeks of treatment, defined as complete response, partial response, or stable disease using RECIST v1.1. The trial had a Simon´s two-stage design, and ≥ 3 of 12 patients with clinical benefit in stage 1 were required to proceed to stage 2. The trial is registered with ClinicalTrials.gov, number NCT03013127. NanoString analysis was performed to explore tumor gene expression signatures and pathways.

Results

Twelve patients were enrolled and received study treatment.

SUBMITTER: Boye K 

PROVIDER: S-EPMC8360887 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature