Implementation of the Time-to-Event Continuous Reassessment Method Design in a Phase I Platform Trial Testing Novel Radiotherapy-Drug Combinations-CONCORDE

JCO Precis Oncol. 2022 Nov:6:e2200133. doi: 10.1200/PO.22.00133.

Abstract

Purpose: CONCORDE is the first phase I drug-radiotherapy (RT) combination platform in non-small-cell lung cancer, designed to assess multiple different DNA damage response inhibitors in combination with radical thoracic RT. Time-to-event continuous reassessment method (TiTE-CRM) methodology will inform dose escalation individually for each different DNA damage response inhibitor-RT combination and a randomized calibration arm will aid attribution of toxicities. We report in detail the novel statistical design and implementation of the TiTE-CRM in the CONCORDE trial.

Methods: Statistical parameters were calibrated following recommendations by Lee and Cheung. Simulations were performed to assess the operating characteristics of the chosen models and were written using modified code from the R package dfcrm.

Results: The results of the simulation work showed that the proposed statistical model setup can answer the research questions under a wide range of potential scenarios. The proposed models work well under varying levels of recruitment and with multiple adaptations to the original methodology.

Conclusion: The results demonstrate how TiTE-CRM methodology may be used in practice in a complex dose-finding platform study. We propose that this novel phase I design has potential to overcome some of the logistical barriers that for many years have prevented timely development of novel drug-RT combinations.

Trial registration: ClinicalTrials.gov NCT04550104.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Carcinoma, Non-Small-Cell Lung* / drug therapy
  • Carcinoma, Non-Small-Cell Lung* / radiotherapy
  • Clinical Trials, Phase I as Topic
  • Drug Combinations
  • Humans
  • Lung Neoplasms* / drug therapy
  • Lung Neoplasms* / radiotherapy
  • Radiation Oncology
  • Randomized Controlled Trials as Topic
  • Research Design

Substances

  • Drug Combinations

Associated data

  • ISRCTN/ISRCTN10142971
  • ClinicalTrials.gov/NCT04550104
  • EudraCT/EudraCT 2020-000206-28