August 15, 2023

Relapsed/refractory AML treatment by Pharmacoscopy-Identified Drugs, a randomized phase-2 clinical trial (PHRT RAPID-01)

8th Call, Clinical Trial

Short Summary

The Snijder Lab at the ETH Zurich has developed a laboratory test to help identify effective treatments for individual AML patients. The platform, called pharmacoscopy, tests the effect of many treatment options on live patient cells in the lab and uses automated microscopy and advanced image analysis to analyze the data. In two separate exploratory clinical studies (DARTT-1 and EXALT-1), the ETH researchers could show that pharmacoscopy might help identify effective treatments for patients with the blood cancer AML.

Goals

The PHRT RAPID-01 Trial will, for the first time in a randomized and controlled clinical trial, evaluate if pharmacoscopy can help clinicians identify effective treatments for AML patients for whom the first-line chemotherapy doesn’t work or stopped working. With Prof. Dr. med. Alexandre Theocharides from the University Hospital Zurich as the lead clinical partner, 88 patients will be enrolled and randomized to either of two trial arms: in the experimental arm, patients will receive pharmacoscopy-guided therapy that is approved for AML, while in the control arm, patients will receive standard-of-care therapies.

Significance

Worldwide, this will be one of the first randomized and controlled clinical trials for treating cancer patients based on such a functional drug testing platform, an approach also called ‘functional precision medicine’. The Cantonal Ethics Commission (KEK) Zurich and Swissmedic have already approved the PHRT RAPID-01 Trial. It is classified as an in vitro diagnostic (IVD) performance trial, with the ETH Zurich as the sponsor and the University Hospital Zurich as the lead clinical partner. The study is performed with support from the Digital Trial Intervention Platform (dTIP) from the ETH Zurich for project management and monitoring, and from the NEXUS Personalized Health Technologies platform for clinical trial statistics. The study is foreseen to open the Inselspital Bern, in collaboration with Prof. Dr. Thomas Pabst, as the second clinical site.

Background

Acute myeloid leukemia (AML) is a rare form of blood cancer affecting around 400 new patients in Switzerland every year. In AML, the rapid growth of abnormal blood cells in the bone marrow and blood interferes with normal blood cell production, severely affecting patients. If left untreated, AML progresses rapidly and is typically fatal within weeks or months. However, even with effective and approved therapies, the prospects for patients for whom the standard first-line chemotherapy doesn’t work are poor, indicating that these patients would benefit from personalized treatments.