Ireland
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Recruiting Phase 1

Eganelisib as Monotherapy and in Combination With Cytarabine in Relapsed/Refractory AML/HR-MDS and in Combination With Azacitidine in Previously Untreated HR-MDS

NCT06533761 · tracked via the Priya Life Science USA tracker
Sponsor
Stelexis BioSciences
Phase
Phase 1
Started
2025-04-28
Last updated
2026-10-20

Condition(s) studied

AML, AdultMDS

Investigational drug(s) / intervention(s)

EganelisibEganelisib in combination with cytarabine →Eganelisib in combination with azacitidine →Azacitadine (AZA) →

Eganelisib: eganelisib will be administered as monotherapy

Eganelisib in combination with cytarabine: eganelisib will be administered in combination with cytarabine

Eganelisib in combination with azacitidine: eganelisib will be administered in combination with azacitidine

Azacitadine (AZA): azacitidine will be administered as standard of care

Study summary

This is a Phase 1b open-label, multicenter, dose-escalation and randomized combination cohort 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) and in combination with azacitidine in PI3K-Gamma-Positive patients with previously untreated HR-MDS.

The study consists of 2 parts:

* Part 1: Dose Escalation (DE) in both monotherapy and in combination with cytarabine.
* Part 2: Randomized Combination Cohort: eganelisib + azacitidine and azacitidine alone

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Part 1: 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. * Part 2: Pathological diagnosis of previously untreated HR-MDS according to WHO 2022 revised criteria, with bone marrow blasts ≥5% and \<20%; Moderate or higher PI3K-Gamma in bone marrow blasts, as determined by an IHC assay; Deemed by the Investigator to require frontline therapy with azacitidine. * 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.

Primary outcome measure(s)

  • 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

Trial sites (12)

FacilityCityRegionStatus
City of Hope Duarte California Recruiting
Anshutz Cancer Pavilion Aurora Colorado Recruiting
Moffitt Cancer Center Tampa Florida Recruiting
Dana-Farber Cancer Institute Boston Massachusetts Recruiting
Washington University in St Louis St Louis Missouri Recruiting
Memorial Sloan Kettering Cancer Center New York New York Recruiting
Montefiore Medical Center New York New York Recruiting
Cleveland Clinic Cleveland Ohio Recruiting
Ohio State University Columbus Ohio Recruiting
MD Anderson Cancer Center Houston Texas Recruiting
Hospital San Pedro de Alcántara Cáceres Spain Recruiting
Hospital Universitari i Politècnic La Fe Valencia Spain Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06533761 on ClinicalTrials.gov ↗ ← All trials in the USA