Eganelisib + Cytarabine for AML

Recruiting at 13 trial locations

Overseen By Clinical Trials Office Stelexis

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Stelexis BioSciences

Must not be taking: Immunosuppressants, Systemic steroids

Disqualifiers: Stem cell transplant, Active infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

125 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for individuals with relapsed or hard-to-treat acute myeloid leukemia (AML) or higher-risk myelodysplastic syndromes (HR-MDS). The study examines the safety and effectiveness of an experimental drug, eganelisib, both alone and in combination with cytarabine. It is suitable for those diagnosed with AML or HR-MDS who have at least 10% cancer cells in their bone marrow. Participants should also maintain a good performance status, meaning they can handle daily activities without much trouble. The trial aims to determine the optimal dose and assess the treatment's effectiveness. As a Phase 1 trial, this research focuses on understanding how the new treatment works in people, offering participants the opportunity to be among the first to receive it.

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop your current medications, but you cannot be on immunosuppressants or systemic steroids unless it's for adrenal insufficiency. If you're on antibiotics, antifungals, or antivirals, you must be stable for over 72 hours before starting the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that eganelisib, when used alone, can significantly slow tumor growth in people with acute myeloid leukemia (AML) and similar conditions. Studies have found it to be safe and generally well-tolerated by patients.

When combined with cytarabine, a common chemotherapy drug, eganelisib might offer a promising treatment for those whose AML has returned or doesn't respond to standard treatments. Early research suggests this combination is safe for humans. However, since this is an early-phase study, the main goal is to determine the best and safest dose for patients.

Please remember that while initial results are positive, the safety and effectiveness of eganelisib, both alone and with cytarabine, remain under thorough investigation. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Eganelisib is unique because it targets the PI3K-gamma pathway, a mechanism that plays a crucial role in immune suppression within the tumor microenvironment. Unlike standard treatments for acute myeloid leukemia (AML), such as chemotherapy drugs like cytarabine and anthracyclines, Eganelisib aims to enhance the body's own immune response against cancer cells. Researchers are excited about this treatment because it offers a novel approach that could potentially improve patient outcomes by making the immune system more effective at fighting the disease. When combined with cytarabine, it may provide a synergistic effect, enhancing the overall treatment efficacy.

What evidence suggests that this trial's treatments could be effective for AML?

Research has shown that eganelisib targets a specific process in leukemia cells by interfering with oxidative phosphorylation, which these cells need to survive and grow. Early lab studies found that eganelisib can greatly reduce the number of leukemia cells. In this trial, some participants will receive eganelisib alone, while others will receive it in combination with cytarabine, a chemotherapy drug. This combination might more effectively slow down or stop cancer cell growth. ^{1 2 6 7 8}

Who Is on the Research Team?

Mieke Ptaszynski, MD

Principal Investigator

Stelexis BioSciences

Are You a Good Fit for This Trial?

This trial is for adults with relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes. Participants must have an ECOG performance status of ≤2, indicating they can perform daily activities with some limitations, and proper liver and kidney function. Those with certain types of AML or less than 10% bone marrow blasts are excluded.

Inclusion Criteria

I am able to get out of my bed or chair and move around.

I have been diagnosed with a specific type of blood cancer, not including acute promyelocytic leukemia, but possibly secondary or treatment-related AML, or high-risk myelodysplastic syndromes.

My liver and kidney functions are within normal ranges.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive escalating doses of eganelisib as monotherapy and in combination with cytarabine to evaluate safety and tolerability

8-12 weeks

Dose Optimization

Participants receive optimized doses of eganelisib and cytarabine to further assess safety and anti-tumor efficacy

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Cytarabine
• Eganelisib

Trial Overview The study tests Eganelisib alone and combined with Cytarabine in two parts: dose escalation to find a safe amount, followed by dose optimization to determine the best therapeutic level. It aims to assess safety, tolerability, how the body processes the drugs (PK), their effects on the disease (PD), and effectiveness against tumors.

How Is the Trial Designed?

Group I: Eganelisib in combination with cytarabine Experimental Treatment 1 Intervention

Group II: Eganelisib Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Stelexis BioSciences

Lead Sponsor

Trials

1

Recruited

130+

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/6008/527688/A-Phase-1b-Open-Label-Study-to-Evaluate-the-Safety

A Phase 1b Open-Label Study to Evaluate the Safety and ... In vitro efficacy studies demonstrated that eganelisib significantly inhibits the viability of multiple leukemic cell lines as well as primary ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06533761

Study Details | NCT06533761 | Eganelisib as Monotherapy ... This is a Phase 1b open-label, multicenter, dose-escalation and dose-optimization study designed to evaluate the safety, tolerability, pharmacokinetics (PK), ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11538868/

Noncanonical PI3Kγ signaling addiction is a therapeutic ... Indeed, targeting PI3Kγ is even more effective than cytarabine in PIK3R5-elevated AML cells without clear side effects, and eganelisib ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2451945624002691

Therapeutic potential of inhibiting the PI3Kγ complex for ... The 5-year overall survival rates for leukemia vary from 90% in acute promyelocytic leukemia (APL) to 65% in B-cell acute lymphocytic leukemia ( ...

5. journals.lww.com journals.lww.com/ijhm/fulltext/2024/13020/expanding_the_therapeutic_horizon_in_acute_myeloid.25.aspx

Expanding the therapeutic horizon in acute myeloid leukemia We demonstrated that inhibition of PI3Kγ with eganelisib disrupts oxidative phosphorylation, thereby impeding AML cell survival and proliferation. Notably, the ...

6. physicianresources.dana-farber.org physicianresources.dana-farber.org/news/dana-farber-researchers-discover-new-target-for-potential-leukemia-therapy

Dana-Farber Researchers Discover New Target for Potential ... "Given what we've observed, we can move very quickly to take these medicines, which appear to be safe and well tolerated, to patients with AML," ...

7. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/523935

Eganelisib as Monotherapy and in Combination With ... This is a Phase 1b open-label, multicenter, dose-escalation and dose-optimization study designed to evaluate the safety, tolerability, ...

8. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-of-eganelisib-alone-and-with-cytarabine-for-patients-with-relapsed-or-refractory-acute-myeloid-leukemia-or-higher-risk-myelodysplastic-syndromes/

Study on the Safety of Eganelisib Alone and with ... This study investigates the safety of Eganelisib, both alone and in combination with Cytarabine, in treating patients suffering from ...