Eganelisib as Monotherapy and in Combination With Cytarabine in Relapsed/Refractory AML

October 20, 2025 updated by: Stelexis BioSciences

A Phase 1b Open-Label Study to Evaluate the Safety and Tolerability of Eganelisib as Monotherapy and in Combination With Cytarabine in Patients With Relapsed/Refractory Acute Myeloid Leukemia

This is a Phase 1b open-label, multicenter, dose-escalation and dose-optimization study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and anti-tumor efficacy of eganelisib as monotherapy and in combination with cytarabine in patients with relapsed/refractory (r/r) acute myeloid leukemia (AML) or r/r higher-risk myelodysplastic syndromes (HR-MDS).

The study consists of 2 parts:

  • Part 1: Dose Escalation (DE) in both monotherapy and in combination.
  • Part 2: Dose Optimization

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

125

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Spain
      • Cáceres, Spain, 10003
        • Recruiting
        • Hospital San Pedro de Alcántara
        • Contact:
      • Valencia, Spain, 46026
        • Recruiting
        • Hospital Universitari i Politecnic La Fe
        • Contact:
  • United States
    • California
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope
        • Contact:
    • Colorado
      • Aurora, Colorado, United States, 80045
    • Florida
      • Tampa, Florida, United States, 33612
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University in St Louis
        • Contact:
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Memorial Sloan Kettering Cancer Center
        • Contact:
      • New York, New York, United States, 10065
        • Not yet recruiting
        • Memorial Sloan Kettering Cancer Center
        • Contact:
      • New York, New York, United States, 10466
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Cleveland Clinic
        • Contact:
          • Ibraheem Al-Aref
          • Phone Number: 216-442-0461
          • Email: ali3@ccf.org
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • Ohio State University
        • Contact:
    • Texas
      • Houston, Texas, United States, 77054
        • Recruiting
        • MD Anderson Cancer Center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Pathological diagnosis of either: AML according to World Health Organization (WHO) 2022 revised criteria per the local pathology report and with ≥10% bone marrow blasts (acute promyelocytic leukemia is excluded but secondary AML and treatment-related AML can be included); Higher-risk (IPSS-R Intermediate, High or Very High Risk at time of study entry) myelodysplastic syndromes (HR-MDS) according to WHO 2022 revised criteria per the local pathology report and with ≥10% bone marrow blasts.
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
  • Adequate hepatic and renal function measured within 7 days prior to the first dose of eganelisib.

Exclusion Criteria:

  • Autologous or allogeneic stem cell transplant within 6 months prior to Cycle 1 Day 1.
  • Receiving immunosuppressants (eg, cyclosporin) or systemic steroids (except for steroid use as cortisol replacement therapy in documented adrenal insufficiency).
  • Active fungal disease or uncontrolled infection of any kind; patients receiving antibiotic, antifungal or antiviral treatment must be afebrile and hemodynamically stable for >72 hours prior to treatment
  • WBC count >25 × 10^9/L measured within 7 days prior to the first dose of eganelisib (hydroxyurea is permitted to decrease the WBC count).
  • Presence of a clinically significant non-hematologic toxicity of prior therapy that has not resolved to Grade ≤1 or Baseline, whichever is worst, as determined by NCI CTCAE v 5.0, except alopecia or skin pigmentation. Fatigue and neuropathy must have resolved to Grade ≤2.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Eganelisib
Drug: Eganelisib
eganelisib will be administered as monotherapy
Experimental: Eganelisib in combination with cytarabine
Drug: Eganelisib in combination with cytarabine
eganelisib will be administered in combination with cytarabine

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence and severity of adverse events (AEs), graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0
Time Frame: 12 months
12 months
Incidence and severity of dose-limiting toxicities (DLTs) in DLT evaluable patients during Cycle 1
Time Frame: 28-35 days
28-35 days
Preliminary clinical activity as evaluated per European LeukemiaNet (ELN) 2022 criteria for AML and International Working Group (IWG) 2023 criteria for HR-MDS
Time Frame: 12 months
12 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Area under the plasma concentration time curve extrapolated to the last measurable time point [AUClast] of eganelisib and cytarabine
Time Frame: 112 days
112 days
Maximum concentration [Cmax] of eganelisib and cytarabine
Time Frame: 112 days
112 days
Time of maximum concentration [Tmax] of eganelisib and cytarabine
Time Frame: 112 days
112 days
Plasma half-life [t1/2] of eganelisib and cytarabine
Time Frame: 112 days
112 days
Measure plasma concentrations of eganelisib and determine model-based PK parameters
Time Frame: 112 days
112 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Stelexis BioSciences

Investigators

  • Study Director: Mieke Ptaszynski, MD, Stelexis BioSciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 28, 2025

Primary Completion (Estimated)

July 15, 2027

Study Completion (Estimated)

March 15, 2028

Study Registration Dates

First Submitted

July 24, 2024

First Submitted That Met QC Criteria

July 30, 2024

First Posted (Actual)

August 1, 2024

Study Record Updates

Last Update Posted (Estimated)

October 22, 2025

Last Update Submitted That Met QC Criteria

October 20, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • STLX-EGA-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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