AML, AdultPhase 1NCT06533761
Eganelisib as Monotherapy and in Combination With Cytarabine in Relapsed/Refractory AML/HR-MDS and in Combination With Azacitidine in Previously Untreated HR-MDS
A Phase 1 study testing Eganelisib, Eganelisib in combination with cytarabine and Eganelisib in combination with azacitidine in people with AML, Adult and MDS, run by Stelexis BioSciences at 12 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML, Adult, MDS
- What is being tested?
- Eganelisib, Eganelisib in combination with cytarabine and Eganelisib in combination with azacitidine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 105 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 28 Apr 2025to 15 Sep 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
12 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 2 countries
United States10 sites, 10 recruiting
- Recruiting
Anshutz Cancer Pavilion
Aurora, Colorado, 80045
Derek Schatz, DEREK.SCHATZ@CUANSCHUTZ.EDU
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Megan Forsyth, 857-215-1405, Megan_Forsyth@DFCI.Harvard.edu
- Recruiting
Cleveland Clinic
Cleveland, Ohio, 44106
Ibraheem Al-Aref, 216-442-0461, ali3@ccf.org
- Recruiting
Ohio State University
Columbus, Ohio, 43210
Sarah Mayne, 614-685-9573, sarah.mayne@osumc.edu
- Recruiting
City of Hope
Duarte, California, 91010
Shukaib Arslan, 626-218-1133, sarslan@coh.org
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77054
Meagan Sachs, 713-503-1589, msachs@mdanderson.org
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Anmol Shah, 646-608-2813, shaha10@mskcc.org
- Recruiting
Montefiore Medical Center
New York, New York, 10466
Davina Hoban, 201-618-4725, davinahoban@montefiore.org
- Recruiting
Washington University in St Louis
St Louis, Missouri, 63110
Alicia Collins, 314-454-8708, aliciac@wustl.edu
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Malko Cajuste, 813-745-0851, Malko.Cajuste@moffitt.org
Spain2 sites, 2 recruiting
- Recruiting
Hospital San Pedro de Alcántara
Cáceres, 10003
Juan Miguel Bergua Burgues, +34 675 699735, jmberguaburg@gmail.com
- Recruiting
Hospital Universitari i Politècnic La Fe
Valencia, 46026
Rebeca Rodriguez Veiga, +34 961 245876, rebeca_rodriguez@iislafe.es
About this study
Official title: 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 or Higher Risk Myelodysplastic Syndrome and Eganelisib in Combination With Azacitidine in PI3K-Gamma-Positive Patients With Previously Untreated Higher Risk Myelodysplastic Syndrome
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
- Conditions
- AML, Adult, MDS
- Being tested
- Eganelisib, Eganelisib in combination with cytarabine, Eganelisib in combination with azacitidine, Azacitadine (AZA)
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EganelisibDrug
eganelisib will be administered as monotherapy
Eganelisib in combination with cytarabineDrug
eganelisib will be administered in combination with cytarabine
Eganelisib in combination with azacitidineDrug
eganelisib will be administered in combination with azacitidine
Azacitadine (AZA)Drug
azacitidine will be administered as standard of care
| Study group | Role | What they receive |
|---|---|---|
| Eganelisib | Experimental | Not described Receives: Drug: Eganelisib |
| Eganelisib in combination with cytarabine | Experimental | Not described Receives: Drug: Eganelisib in combination with cytarabine |
| Eganelisib in combination with azacitidine | Experimental | Not described Receives: Drug: Eganelisib in combination with azacitidine |
| Azacitidine | Active comparator | Not described Receives: Drug: Azacitadine (AZA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
Incidence and severity of dose-limiting toxicities (DLTs) in DLT evaluable patients during Cycle 1
Measured: 28-35 days
Secondary outcomes (6)
Preliminary clinical activity as evaluated per European LeukemiaNet (ELN) 2022 criteria for AML and International Working Group (IWG) 2023 criteria for HR-MDS and FDA 2025 draft guidance
Measured: 12 months
Area under the plasma concentration time curve extrapolated to the last measurable time point [AUClast] of eganelisib, cytarabine, and azacitidine
Measured: 112 days
Maximum concentration [Cmax] of eganelisib, cytarabine, and azacitidine
Measured: 112 days
Time of maximum concentration [Tmax] of eganelisib, cytarabine, and azacitidine
Measured: 112 days
Plasma half-life [t1/2] of eganelisib, cytarabine, and azacitidine
Measured: 112 days
Measure plasma concentrations of eganelisib and determine model-based PK parameters
Measured: 112 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2024
- Study start28 Apr 2025
- Primary completion15 Dec 2027 (estimated)
- Study completion15 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, AdultStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 105 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stelexis BioSciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06533761
- Sponsor's protocol ID
- STLX-EGA-001
- Status last verified
- Oct 2026
- First submitted
- 24 Jul 2024
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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 or Higher Risk Myelodysplastic Syndrome and Eganelisib in Combination With Azacitidine in PI3K-Gamma-Positive Patients With Previously Untreated Higher Risk Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT06533761
- Acronym / sponsor ID
- STLX-EGA-001
- Conditions
- AML, Adult, MDS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 105 participants (estimated)
- Lead sponsor
- Stelexis BioSciencesIndustry (pharma, biotech, devices)
- Study start
- 28 Apr 2025 (actual)
- Primary completion
- 15 Dec 2027 (estimated)
- Study completion
- 15 Sep 2028 (estimated)
- First posted
- 1 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Eganelisib | Experimental | Drug: Eganelisib |
| Eganelisib in combination with cytarabine | Experimental | Drug: Eganelisib in combination with cytarabine |
| Eganelisib in combination with azacitidine | Experimental | Drug: Eganelisib in combination with azacitidine |
| Azacitidine | Active comparator | Drug: Azacitadine (AZA) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Eganelisibeganelisib will be administered as monotherapy | Drug | Eganelisib |
| Eganelisib in combination with cytarabineeganelisib will be administered in combination with cytarabine | Drug | Eganelisib in combination with cytarabine |
| Eganelisib in combination with azacitidineeganelisib will be administered in combination with azacitidine | Drug | Eganelisib in combination with azacitidine |
| Azacitadine (AZA)azacitidine will be administered as standard of care | Drug | Azacitidine |
Primary outcome
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
Incidence and severity of dose-limiting toxicities (DLTs) in DLT evaluable patients during Cycle 1
Time frame: 28-35 days
6 secondary outcomes
Preliminary clinical activity as evaluated per European LeukemiaNet (ELN) 2022 criteria for AML and International Working Group (IWG) 2023 criteria for HR-MDS and FDA 2025 draft guidance
Time frame: 12 months
Area under the plasma concentration time curve extrapolated to the last measurable time point [AUClast] of eganelisib, cytarabine, and azacitidine
Time frame: 112 days
Maximum concentration [Cmax] of eganelisib, cytarabine, and azacitidine
Time frame: 112 days
Time of maximum concentration [Tmax] of eganelisib, cytarabine, and azacitidine
Time frame: 112 days
Plasma half-life [t1/2] of eganelisib, cytarabine, and azacitidine
Time frame: 112 days
Measure plasma concentrations of eganelisib and determine model-based PK parameters
Time frame: 112 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Eganelisib, Eganelisib in combination with cytarabine and Eganelisib in combination with azacitidine in people with AML, Adult and MDS, run by Stelexis BioSciences at 12 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials Office Stelexis (508-543-6979, clinicaltrials@stelexis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2026.