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

Phase 1

Recruiting

• Conditions: MDS; AML, Adult
• Interventions: Drug: Eganelisib; Drug: Eganelisib in combination with cytarabine

• Registration Number: NCT06533761
• Lead Sponsor: Stelexis BioSciences

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-07-24
• Study First Submitted QC: 2024-07-30
• Study First Posted: 2024-08-01
• Study Start: 2025-04-28
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-13
• Last Update Posted: 2025-05-16
• Primary Completion: 2027-07-15
• Study Completion: 2028-03-15

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 125

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Eganelisib	Eganelisib	-
Eganelisib in combination with cytarabine	Eganelisib in combination with cytarabine	-

Primary Outcome Measures

Name	Time	Method
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
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

Secondary Outcome Measures

Name	Time	Method
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
Area under the plasma concentration time curve extrapolated to the last measurable time point [AUClast] of eganelisib and cytarabine	112 days

Trial Locations

Locations (1)

Montefiore Medical Center; 🇺🇸 New York, New York, United States