Dataset from A Phase I Open-label, Dose Escalation Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of GSK2879552 Given Orally in Subjects With Relapsed/Refractory Acute Myeloid Leukemia

This study is a phase I, open-label study to determine recommended phase 2 dose (RP2D) and
regimen for the orally administered lysine specific demethylase 1 (LSD1) inhibitor
GSK2879552, alone or in combination with All-Trans Retinoic Acid (ATRA). The recommended dose
and regimen will be selected based on the safety, pharmacokinetic (PK), and pharmacodynamic
(PD) profiles observed after the treatment of subjects with relapsed/refractory AML. The
study consists of two parts. Part 1 will identify the maximum tolerated dose (MTD) and/or
RP2D using a dose-escalation procedure. Dose escalations will be guided by the
Neuenschwander-continual reassessment method (N-CRM). PK/PD expansion cohorts will also be
included in Part 1 to characterize the range of biologically effective doses by assessing PD
markers and obtain additional PK data. Part 2 will explore further the safety, tolerability,
and clinical activity of GSK2879552, alone or in combination with ATRA, at the RP2D in
subjects with AML.