Using eganelisib alone and with cytarabine for treating 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

This study is testing if a new drug called eganelisib, used alone or with another drug called cytarabine, can help people with relapsed or hard-to-treat acute myeloid leukemia feel better.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 125 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Stelexis BioSciences (industry)
• Locations: 13 sites (Duarte, California and 12 other locations)
• Trial ID: NCT06533761 on ClinicalTrials.gov

What this trial studies

This Phase 1b open-label study evaluates the safety and effectiveness of eganelisib, both as a standalone treatment and in combination with cytarabine, for patients with relapsed or refractory acute myeloid leukemia (AML) and higher-risk myelodysplastic syndromes (HR-MDS). The study is divided into two parts: a dose escalation phase to determine the appropriate dosage and a dose optimization phase to refine treatment protocols. It aims to assess pharmacokinetics, pharmacodynamics, and anti-tumor efficacy in a multicenter setting.

Who should consider this trial

Good fit: Ideal candidates include adults with relapsed or refractory AML or higher-risk MDS who have a specific percentage of bone marrow blasts.

Not a fit: Patients who have undergone stem cell transplants recently or are currently on immunosuppressants may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat forms of AML and HR-MDS.

How similar studies have performed: While this approach is novel, similar studies have shown promise in targeting AML with combination therapies.

Eligibility criteria

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.

Where this trial is running

Duarte, California and 12 other locations

• City of Hope — Duarte, California, United States (RECRUITING)
• Anshutz Cancer Pavilion — Aurora, Colorado, United States (RECRUITING)
• Moffitt Cancer Center — Tampa, Florida, United States (RECRUITING)
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States (RECRUITING)
• Washington University in St Louis — St Louis, Missouri, United States (RECRUITING)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (NOT_YET_RECRUITING)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (RECRUITING)
• Montefiore Medical Center — New York, New York, United States (RECRUITING)
• Cleveland Clinic — Cleveland, Ohio, United States (RECRUITING)
• Ohio State University — Columbus, Ohio, United States (RECRUITING)
• MD Anderson Cancer Center — Houston, Texas, United States (RECRUITING)
• Hospital San Pedro de Alcántara — Cáceres, Spain (RECRUITING)
• Hospital Universitari i Politècnic La Fe — Valencia, Spain (RECRUITING)

Study contacts

• Study coordinator: Clinical Trials Office Stelexis

Conditions: AML, Adult, MDS