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

  • End date
    Feb 8, 2027
  • participants needed
  • sponsor
    University of Colorado, Denver
Updated on 7 October 2022
direct bilirubin
gilbert's syndrome
refractory acute myeloid leukemia (aml)


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


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.

Condition Acute Myeloid Leukemia, Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia Refractory
Treatment KPT-9274
Clinical Study IdentifierNCT04914845
SponsorUniversity of Colorado, Denver
Last Modified on7 October 2022


Yes No Not Sure

Inclusion Criteria

Written informed consent obtained prior to any study related procedures required solely for this research study
Age ≥18 years
Patients with WHO-confirmed non-APL AML who have not responded to or relapsed after at least one prior therapy and for whom no standard therapy that may provide clinical benefit is available
Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2
Adequate hepatic function
Total bilirubin < 1.5 times the upper limit of normal (ULN) (except patients
with Gilbert's syndrome [hereditary indirect hyperbilirubinemia]; in the case
of Gilbert's syndrome the direct bilirubin must be ≤2.0 times the ULN)
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5
times ULN (except patients with known liver involvement of their AML who must
have AST and ALT ≤ 5.0 times ULN)
Adequate renal function: estimated creatinine clearance of ≥ 60 mL/min, calculated using the formula of Cockroft and Gault (140-Age) • Mass (kg)/(72 • creatinine mg/dL); multiply by 0.85 if female
Female patients of child-bearing potential must agree to use dual methods of contraception (including one highly effective and one effective method of contraception) and have a negative serum pregnancy test at Screening. For both male and female patients, effective methods of contraception must be used throughout the study and for 3 months following the last dose
Male patients must use an effective barrier method of contraception if sexually active with a female of child-bearing potential. For both male and female patients, effective methods of contraception must be used throughout the study and for 3 months following the last dose

Exclusion Criteria

Radiation, chemotherapy, immunotherapy or any other anticancer therapy, including investigational anti-cancer therapy ≤ 2 weeks prior to C1D1. Hydroxyurea is not considered an anti-cancer therapy
White blood cell count ≥25x109/L (hydroxyurea or leukapheresis permitted to reduce to below the exclusion criteria threshold and allow eligibility)
Female patients who are pregnant or lactating
Patients who have not recovered or stabilized (Grade 1 or to their baseline for non-hematologic toxicities) from toxicities related to their previous treatment, except for alopecia
Patients with known active central nervous system (CNS) disease
Clinically significant severe heart disease
Subjects with uncontrolled systemic fungal, bacterial, viral or other infection despite appropriate antibiotics or other treatment
Known, active hepatitis A, B, or C infection; or known to be positive for HCV RNA or HBsAg (HBV surface antigen). Testing is not required
Patients with significantly diseased or obstructed gastrointestinal tract or uncontrolled vomiting or diarrhea that could interfere with the absorption of KPT-9274
Serious psychiatric or medical conditions that, in the opinion of the Investigator, could interfere with treatment, compliance, or the ability to give consent
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