AB8939 for Acute Myeloid Leukemia

Recruiting at 10 trial locations

Overseen By Clinical Study Coordinator

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: AB Science

Disqualifiers: Standard care, HSCT eligible, CNS leukemia, others

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

Compensation: Varies

Number of Visits: Unknown

78 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AB8939 for people with acute myeloid leukemia (AML), a type of blood cancer. The goal is to determine the safety and effectiveness of this treatment, both alone and in combination with another drug, venetoclax. The study seeks individuals diagnosed with AML who are looking for additional treatment options after initial treatments. Participants should be comfortable with study procedures, such as bone marrow biopsies, and should not qualify for standard treatments or specific types of transplants. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Research has shown that the combination of AB8939 and Venetoclax appears safe. In an early study, all three patients with acute myeloid leukemia (AML) responded well to this treatment, achieving a 100% disease control rate as their disease stopped progressing. No serious side effects were reported in this small group, suggesting the treatment might be manageable for patients.

For AB8939 alone, the treatment remains under study. In one case, a patient experienced a significant reduction in cancer cells in their bone marrow after receiving a lower dose. Detailed reports of serious side effects for this treatment are not yet available, but ongoing trials aim to gather more information about its safety.

These findings are promising, but more research is needed to fully understand the safety and effectiveness of both treatments. The trial under consideration aims to explore this further. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about AB8939 for Acute Myeloid Leukemia (AML) because it uses a new mechanism of action that sets it apart from current treatments like chemotherapy and targeted therapies. AB8939 is designed to disrupt the microtubule structure within cancer cells, which is crucial for cell division and survival, offering a novel approach to combating the disease. Moreover, one arm of the trial combines AB8939 with Venetoclax, a treatment that targets and inhibits the BCL-2 protein, potentially enhancing the overall effectiveness. This combination could provide a powerful one-two punch against AML, addressing both cell division and apoptosis pathways.

What evidence suggests that this trial's treatments could be effective for acute myeloid leukemia?

Research has shown that AB8939 holds promise for treating acute myeloid leukemia (AML). In studies, AB8939 significantly reduced leukemia cells in patients. For instance, one patient experienced a drop in immature blood cells in the bone marrow from 55% to 5% within a month. In this trial, some participants will receive AB8939 alone, while others will receive it combined with venetoclax. When combined with venetoclax, all three patients in a small group responded positively, with one achieving complete remission. These early results suggest that AB8939, both alone and with venetoclax, could be effective for AML. ^{1 3 6 7 8}

Who Is on the Research Team?

Norbert Vey, MD

Principal Investigator

Institut Paoli Calmettes, Marseille, France

Nicholas Short, MD

Principal Investigator

MD Anderson Cancer Center, Houston, Texas

Are You a Good Fit for This Trial?

This trial is for adults with Acute Myeloid Leukemia or high-risk Myelodysplastic Syndromes who've had previous treatments fail. They must be in good physical condition (ECOG ≤ 1), able to consent, and follow study procedures like bone marrow biopsies. It's not for those eligible for standard care, stem cell transplant, have certain leukemia types or CNS involvement, recent transplants, or are pregnant/breastfeeding.

Inclusion Criteria

I have acute myeloid leukemia and need second or third line treatment.

I am fully active and can carry on all my pre-disease activities without restriction.

I have a high-risk myelodysplastic syndrome not responding to first treatments.

Exclusion Criteria

I am eligible for a stem cell transplant.

I am currently pregnant.

I have active leukemia in my brain or spinal cord.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Dose escalation study to determine safety, tolerability, and pharmacokinetic profiles of AB8939

8 weeks

Multiple visits (in-person)

Dose Expansion

Dose expansion study to determine the schedule for Phase 2 and provide early efficacy assessment

8 weeks

Multiple visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AB8939

Trial Overview The trial tests AB8939's safety and tolerability in patients with AML by finding the highest dose they can take without serious side effects. Participants will also receive Azacitidine as part of the treatment regimen.

How Is the Trial Designed?

Group I: AB8939 plus Venetoclax Experimental Treatment 2 Interventions

AB8939 administered in combination with venetoclax

Group II: AB8939 Experimental Treatment 1 Intervention

AB8939 administered as a single agent

Who Is Running the Clinical Trial?

AB Science

Lead Sponsor

Trials

39

Recruited

15,700+

Citations

1. biospace.com biospace.com/press-releases/ab-science-provides-intitial-phase-1-data-for-the-combination-of-ab8939-with-venetoclax-for-the-treatment-of-refractory-or-relapsed-acute-myeloid-leukemia

AB Science provides intitial Phase 1 data for the ... The disease control rate was 100% (3/3), and the partial response rate is 100% (3/3), including one patient in complete remission. Two of the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05211570?cond=GATA2%20deficiency&rank=9

Study Details | NCT05211570 | AB8939 in Patients With ... This dose expansion study aims to determine the schedule for a Phase 2 trial in patients with relapsed/refractory AML and to also provide an early efficacy ...

3. rarecancernews.com rarecancernews.com/blood-cancer/all-3-patients-high-risk-aml-see-benefit-combination-treatment-trial/

3 patients in trial to date see benefits with treatment for AML When combined with Venclexta, early results indicated positive responses after the first AB8939 cycle — 14 days of treatment — in AML patients ...

4. ab-science.com ab-science.com/wp-content/uploads/2024/12/CP-AB8939-Update-16122024-VENG-VF.pdf

PRESS RELEASE - AB Science AB8939 has activity seen across refractory AML cell lines, has an additive effect with referenced first line treatment for AML, namely ...

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40884567/

a meta-analysis of clinical trials and Real-World outcomes In real-world studies, the VEN + HMA combination was associated with a significantly higher CRc rate (67%, 95% CI: 48-85%) compared to HMA ...

6. ab-science.com ab-science.com/pipeline/ab8939-acute-myeloid-leukemia/

AB8939 – Acute Myeloid Leukemia - AB Science AB Science is planning human clinical trial of AB8939 to investigate its tolerability and efficacy in patients with acute myeloid leukemia.

7. targetedonc.com targetedonc.com/view/complete-bone-response-observed-in-trial-of-ab8939-for-aml

Complete Bone Response Observed in Trial of AB8939 for ... The patient had a reduction from 55% to 5% in bone marrow blast cells 1 month after receiving the second-lowest dose increment used in the trial ...

8. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-ab8939-and-azacitidine-for-patients-with-relapsed-or-refractory-acute-myeloid-leukemia/

Study on the Safety and Effects of AB8939 and Azacitidine ... This study tests the safety and efficacy of AB8939 and Azacitidine in treating patients with relapsed or refractory Acute Myeloid Leukemia and ...