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PSI Conference 2019: Preliminary Design of a Basket Trial for Cancer Cachexia

04 June 2019

Cachexia is a metabolic disorder that occurs with several chronic diseases including cancer, heart failure, chronic obstructive pulmonary disease, and chronic kidney disease. Cachexia manifests as marked involuntary body weight loss, muscle atrophy, and reduced appetite, progressing to significant functional impairment and increased risk of death. Traditionally, cancer clinical trials have focused on testing a compound in patients with a single tumour type, e.g. breast cancer. Becoming increasingly used, however, are basket trials, where a compound is tested against a single mutation across a number of tumour types (i.e. assuming that it is the mutation that is important, not the location of the tumour). Pfizer’s early clinical development portfolio currently includes a project targeting cancer cachexia. The project has been investigating the possibility of utilising a basket trial design for its First-in-Patient study. A preliminary design for such a trial, looking at lean body mass in patients with solid tumours and cancer cachexia, will be presented. The design incorporates an interim analysis to assess both the heterogeneity of treatment effects across the different tumour types, as well as the likelihood of end-of-study success using Bayesian predictive probabilities.

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