Eganelisib as Monotherapy and in Combination With Cytarabine in Relapsed/Refractory AML
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
1 other identifier
interventional
125
2 countries
13
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Apr 2025
Typical duration for phase_1
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 24, 2024
CompletedFirst Posted
Study publicly available on registry
August 1, 2024
CompletedStudy Start
First participant enrolled
April 28, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 15, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 15, 2028
October 22, 2025
October 1, 2025
2.2 years
July 24, 2024
October 20, 2025
Conditions
Outcome Measures
Primary Outcomes (3)
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
12 months
Incidence and severity of dose-limiting toxicities (DLTs) in DLT evaluable patients during Cycle 1
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
12 months
Secondary Outcomes (5)
Area under the plasma concentration time curve extrapolated to the last measurable time point [AUClast] of eganelisib and cytarabine
112 days
Maximum concentration [Cmax] of eganelisib and cytarabine
112 days
Time of maximum concentration [Tmax] of eganelisib and cytarabine
112 days
Plasma half-life [t1/2] of eganelisib and cytarabine
112 days
Measure plasma concentrations of eganelisib and determine model-based PK parameters
112 days
Study Arms (2)
Eganelisib
EXPERIMENTALEganelisib in combination with cytarabine
EXPERIMENTALInterventions
eganelisib will be administered in combination with cytarabine
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (13)
City of Hope
Duarte, California, 91010, United States
Anshutz Cancer Pavilion
Aurora, Colorado, 80045, United States
Moffitt Cancer Center
Tampa, Florida, 33612, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Washington University in St Louis
St Louis, Missouri, 63110, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Memorial Sloan Kettering Cancer Center
New York, New York, 10065, United States
Montefiore Medical Center
New York, New York, 10466, United States
Cleveland Clinic
Cleveland, Ohio, 44106, United States
Ohio State University
Columbus, Ohio, 43210, United States
MD Anderson Cancer Center
Houston, Texas, 77054, United States
Hospital San Pedro de Alcántara
Cáceres, 10003, Spain
Hospital Universitari i Politècnic La Fe
Valencia, 46026, Spain
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 24, 2024
First Posted
August 1, 2024
Study Start
April 28, 2025
Primary Completion (Estimated)
July 15, 2027
Study Completion (Estimated)
March 15, 2028
Last Updated
October 22, 2025
Record last verified: 2025-10
Data Sharing
- IPD Sharing
- Will not share