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

NCT04914845 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-01-27

Study results available
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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.

Conditions

Interventions

DRUG

KPT-9274

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.

Sponsors & Collaborators

  • Karyopharm Therapeutics Inc

    collaborator INDUSTRY
  • University of Colorado, Denver

    lead OTHER

Principal Investigators

  • Daniel Pollyea, MD · University of Colorado, Denver

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-08-27
Primary Completion
2025-07-08
Completion
2025-10-22
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04914845 on ClinicalTrials.gov