Reduced Venetoclax for Acute Myeloid Leukemia

Overseen By Lalta Dhanantwari, MBA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Northwell Health

Must be taking: Hypomethylating agents

Must not be taking: Targeted therapies, Investigational drugs

Disqualifiers: APL, CNS involvement, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 1

Duration: 12 months

41 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a lower dose of Venetoclax, combined with either Azacitidine or Decitabine, to determine its effectiveness in treating acute myeloid leukemia (AML), a type of blood cancer. It targets individuals previously treated for AML who have reached a stage where cancer cells are no longer visible under a microscope. Participants should have AML, have undergone specific initial therapies, and show signs of cancer remission without recent transfusions or growth factor support. The study aims to discover if a reduced dose can maintain leukemia control while potentially causing fewer side effects. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot be on certain targeted therapies or investigational drugs. It's best to discuss your specific medications with the trial team.

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

Research has shown that using azacitidine and venetoclax together is generally safe. Patients in everyday medical practice tolerated this combination well, even at lower doses, with a lower chance of serious side effects compared to stronger treatments.

Similarly, studies on the combination of decitabine and venetoclax have shown promising safety results. For young patients with acute myeloid leukemia, this combination demonstrated a high safety profile, with fewer serious side effects.

Both combinations have proven safe for different patient groups, suggesting that the treatment might also be well-tolerated in clinical trials. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about Venetoclax for treating Acute Myeloid Leukemia (AML) because it targets a protein called BCL-2, which helps cancer cells survive. Most current AML treatments, like chemotherapy, attack rapidly dividing cells non-specifically, which can lead to significant side effects. Venetoclax, on the other hand, specifically induces cancer cell death by disrupting the survival signals, potentially offering a more targeted and less toxic approach. Additionally, when combined with drugs like Azacitidine or Decitabine, Venetoclax might enhance the effectiveness of these standard treatments, offering new hope for better outcomes.

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

Research has shown that combining azacitidine and venetoclax, one of the treatment arms in this trial, holds promise for treating acute myeloid leukemia (AML). In several studies, more than half of the patients experienced remission, meaning their cancer was no longer detectable. On average, patients lived about 16.9 months after starting this treatment. Similarly, the combination of decitabine and venetoclax, another treatment arm in this trial, proved effective, with one study showing a 93% response rate in young adults. These findings suggest both treatment combinations could effectively manage AML. ^{1 2 4 5 6}

Who Is on the Research Team?

Boiclair Stephanie, MD

Principal Investigator

Northwell Health

Are You a Good Fit for This Trial?

This trial is for AML patients in remission who can't undergo intensive treatment. They must have completed initial therapy with Venetoclax and a hypomethylating agent (HMA) without severe side effects or disease progression.

Inclusion Criteria

Consent to be obtained within a specific timeframe of bone marrow biopsy report showing morphological remission

Patient must be able to understand and sign informed consent and additional study documents

I can take pills and will follow the study plan.

Exclusion Criteria

Treatment with another investigational drug

I haven't used growth factor injections in the last 14 days.

I am currently taking medication for cancer that targets specific genes.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Venetoclax for 14 days per cycle along with a hypomethylating agent, with cycles occurring every 28 days

12 months

Visit on Day 1 of each cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Extension/Long-term follow-up

Participants may continue to be monitored for event-free survival and other outcomes

48 months

What Are the Treatments Tested in This Trial?

Interventions

• Venetoclax

Trial Overview The study tests if taking Venetoclax for only 14 days per cycle, along with an HMA, maintains remission in AML patients while reducing serious blood-related side effects compared to longer exposure times.

How Is the Trial Designed?

Group I: Azacytadine + Venetoclax Active Control 2 Interventions

Group II: Decitabine + Venetoclax Active Control 2 Interventions

Who Is Running the Clinical Trial?

Northwell Health

Lead Sponsor

Trials

481

Recruited

470,000+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40951718/

Real-world outcomes of azacitidine plus venetoclax in ... After treatment, more than half went into remission (the cancer was no longer detectable), and those with newly diagnosed AML had better ...

2. nature.com nature.com/articles/s41375-025-02730-3

Outcomes of patients treated with venetoclax plus ... Efficacy, including composite complete remission and overall survival, were improved with venetoclax plus azacitidine vs. placebo plus ...

3. nejm.org nejm.org/doi/full/10.1056/NEJMoa2012971

Azacitidine and Venetoclax in Previously Untreated Acute ... At a median follow-up of 14.9 months, the median overall survival was 16.9 months. This confirmatory trial (VIALE-A) was designed to evaluate ...

4. hematologyadvisor.com hematologyadvisor.com/news/combination-azacitidine-venetoclax-acute-myeloid-leukemia-aml-treatment-risk/

Combination Azacitidine, Venetoclax Effective in AML ... Analysis showed that the overall response rates in the azacitidine plus venetoclax, chemotherapy, and azacitidine monotherapy groups were 38.5%, ...

5. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.e18509

Comparative safety and effectiveness of azacitidine plus ... In AML patients aged 60–75 years, HMA&Ven demonstrated comparable all-cause mortality to IC and significantly lower risk of several adverse events.

6. ashpublications.org ashpublications.org/ashclinicalnews/news/8514/A-Study-Evaluating-Safety-and-Efficacy-of

A Study Evaluating Safety and Efficacy of Venetoclax in ... A study evaluating safety and efficacy of venetoclax in combination with azacitidine versus standard of care after allogeneic stem cell transplant.