Study design and oversights
Cancer
CANCER
We conducted a phase 1, multicentre, open-label, dose-escalation study (ClinicalTrials.gov, NCT04065399). Revumenib was administered orally in either capsule or liquid formulation, q12h, in 28-day continuous cycles with dose adjustment by body surface area for patients weighing under 40 kg (Extended Data Fig. The study was conducted in accordance with the principles of the Declaration of Helsinki and the International Council for Harmonization Good Clinical Practice. The protocol and amendments were approved by the institutional review board or ethics committee at The University of Texas MD Anderson Cancer Center, City of Hope, Washington University School of Medicine in St. Louis, Dana-Farber Cancer Institute, Winship Cancer Institute, Emory University School of Medicine, University of Chicago, Florida Cancer Specialists/Sarah Cannon Research Institute, University of Iowa and Memorial Sloan Kettering Cancer Center. All patients provided written informed consent. This study was designed by the sponsor (Syndax Pharmaceuticals) in collaboration with the investigators. The data were collected by the investigators and their research staff and were analysed by the sponsor and authors. Drafts of the manuscript were written by the first and last authors and revised in collaboration with all authors and the sponsor, all of whom vouch for the completeness and accuracy of the data and analyses, and for the adherence of the study to the protocol. Assistance in manuscript preparation for submission was provided by a professional service and paid for by the sponsor.