Spots Global Cancer Trial Database for KPT-9274 in Patients With Relapsed and Refractory Acute Myeloid Leukemia

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Trial Identification

Brief Title: KPT-9274 in Patients With Relapsed and Refractory Acute Myeloid Leukemia

Official Title: A Phase 1 Open-label Study of KPT-9274 in Patients With Relapsed and Refractory Acute Myeloid Leukemia

Study ID: NCT04914845

Conditions

Acute Myeloid Leukemia

Acute Myeloid Leukemia, in Relapse

Acute Myeloid Leukemia Refractory

Interventions

KPT-9274

Study Description

Brief Summary: This study will evaluate the safety and tolerability of oral KPT-9274 for the treatment of patients with relapsed or refractory acute myeloid leukemia.

Detailed Description: KPT-9274 is a first-in-class orally bioavailable, non-competitive, small molecule, dual modulator of p21 protein (Cdc42/Rac)-activated kinase 4 (PAK4) and nicotinamide phosphoribosyltransferase /PBEF/visfatin (NAMPT). NAMPT is the rate-limiting enzyme in the metabolic scavenging pathway that utilizes nicotinamide to replenish nicotinamide adenine dinucleotide (NAD), an essential metabolic cofactor and second messenger. Recent investigations have shown that in in vitro and in vivo models, NAMPT is uniquely essential for relapsed or refractory AML stem cells. Targeting relapsed AML stem cells, through targeting of NAMPT, may allow for a promising therapeutic opportunity for patients with relapsed or refractory AML.