Ziftomenib for AML

Recruiting at 1 trial location

Overseen By KO-MEN-017 Clinical Operations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Kura Oncology, Inc.

Disqualifiers: Prior AML therapy, APL, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

1300 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of a new drug, ziftomenib, in treating acute myeloid leukemia (AML), a type of blood cancer. Researchers aim to determine if adding ziftomenib to standard AML treatments benefits patients with specific genetic changes in their cancer cells. The trial includes two separate studies: one for older or less healthy patients using a gentler treatment approach, and another for patients who can handle more intense treatment. This trial may suit individuals who have not yet received treatment for AML and have certain genetic markers in their cancer. Participants will not know if they are receiving ziftomenib or a placebo, as the study is double-blinded. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Do I have to stop taking my current medications for the Ziftomenib for AML trial?

The trial information does not specify if you need to stop taking your current medications. However, since the trial involves adding ziftomenib to standard AML treatments, it's best to discuss your current medications with the trial team to ensure there are no interactions.

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

Research has shown that ziftomenib is generally safe for patients with AML (acute myeloid leukemia) when used with standard treatments. In studies combining ziftomenib with venetoclax and azacitidine, patients tolerated the treatment well at the tested doses. The results also suggested effectiveness, with no serious safety issues identified.

Another study used ziftomenib with 7+3 chemotherapy (cytarabine and daunorubicin). This combination achieved a high success rate and was well-tolerated. Safety remained consistent across different doses, with no dangerous side effects observed.

These findings suggest that ziftomenib, when used with either treatment combination, maintains a strong safety record in similar patient groups. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment for AML?

Researchers are excited about Ziftomenib for acute myeloid leukemia (AML) because it offers a novel approach by targeting a specific mutation that current treatments may not address effectively. Unlike the standard options like chemotherapy, which broadly attack dividing cells, Ziftomenib focuses on inhibiting the menin-MLL interaction, a key driver in certain AML subtypes. This targeted mechanism could lead to more effective treatment with potentially fewer side effects. Additionally, Ziftomenib is being tested in combination with existing drugs like venetoclax and azacitidine, which could enhance its efficacy and offer new hope for patients who have limited options with traditional therapies.

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

Research has shown that ziftomenib could help treat acute myeloid leukemia (AML) with specific genetic changes. In this trial, participants in the nonintensive therapy study may receive ziftomenib with venetoclax and azacitidine, which previous studies have shown to significantly improve outcomes. Another arm of this trial uses ziftomenib with cytarabine and daunorubicin, common AML treatments. In earlier studies, 94% of patients experienced a reduction in cancer symptoms with this combination. These results suggest that ziftomenib might be a useful addition to current AML treatments. ^{1 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults with acute myeloid leukemia (AML) who have specific genetic mutations (NPM1-m or KMT2A-r), are FLT3 wild-type, and haven't been treated before. They must be over 18, in fairly good health (ECOG 0-2), and if they can have children, agree to use contraception. People with certain heart conditions or severe liver/kidney issues may not qualify.

Inclusion Criteria

I am 18 years old or older.

I have been diagnosed with AML according to the latest WHO guidelines.

I am able to care for myself and up to being able to work.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment - Nonintensive Therapy Study

Participants receive venetoclax and azacitidine, plus either ziftomenib or a placebo

Up to 36 months

Treatment - Intensive Therapy Study

Participants receive cytarabine and daunorubicin (induction), cytarabine (consolidation), and either ziftomenib or a placebo (maintenance)

Up to 36 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ziftomenib

Trial Overview The trial tests Ziftomenib combined with standard AML treatments: Venetoclax + Azacitidine for older/sicker patients, or Cytarabine + Daunorubicin for fitter patients. It's a double-blind study where neither doctors nor participants know if they're getting Ziftomenib or a placebo alongside their cancer treatment.

How Is the Trial Designed?

Group I: Nonintensive Therapy Study, Arm A Experimental Treatment 3 Interventions

Ziftomenib in combination with venetoclax+azacitidine

Group II: Intensive Therapy Study, Arm B Experimental Treatment 4 Interventions

Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), placebo (maintenance)

Group III: Intensive Therapy Study, Arm A Experimental Treatment 3 Interventions

Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), ziftomenib (maintenance)

Group IV: Nonintensive Therapy Study, Arm B Placebo Group 3 Interventions

Placebo in combination with venetoclax+azacitidine

Group V: Intensive Therapy Study, Arm C Placebo Group 3 Interventions

Placebo+cytarabine+daunorubicin (induction), placebo+cytarabine (consolidation), placebo (maintenance)

Who Is Running the Clinical Trial?

Kura Oncology, Inc.

Lead Sponsor

Trials

19

Recruited

1,700+

Citations

1. onclive.com onclive.com/view/ziftomenib-with-7-3-shows-early-promise-in-newly-diagnosed-npm1-mutated-kmt2a-rearranged-aml

Ziftomenib With 7+3 Shows Early Promise in Newly ... Ziftomenib combined with 7+3 chemotherapy shows a 94% objective response rate in newly diagnosed NPM1-mutated and KMT2A-rearranged AML patients.

2. kyowakirin.com kyowakirin.com/media_center/news_releases/2025/pdf/e20250612_01.pdf

Kura Oncology and Kyowa Kirin Report Positive Updated ... In the ongoing study, ziftomenib dosed once daily at 600 mg in combination with 7+3 continued to demonstrate robust and evolving clinical ...

3. ir.kuraoncology.com ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-launch-clinical-trial-evaluating

Kura Oncology and Kyowa Kirin Launch Clinical Trial ... This cohort evaluates ziftomenib, a once-daily, investigational oral menin inhibitor, combined with cytarabine and daunorubicin (7+3) as well as ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12175398/

clinical trial updates from 2024 ASH annual meeting Meanwhile, ziftomenib combined with venetoclax and azacitidine (NCT05735184) produced 50% (24/48) ORR and 25% (12/48) CR/CRh rate in KMT2Ar/ ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT07007312

NCT07007312 | Studies to Assess Ziftomenib in ... Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations.

6. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/214/530335/Ziftomenib-Combined-with-Intensive-Induction-7-3

Ziftomenib Combined with Intensive Induction (7+3) in Newly ... Conclusion: In the ongoing KOMET-007 study, ziftomenib combined with 7+3 was well tolerated, with a consistent safety profile across dose levels ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT05735184

NCT05735184 | A Study to Investigate the Safety and ... The purpose of this study is to assess the safety, tolerability, and early signs of efficacy of ziftomenib in combination with SOC drugs to treat AML. Official ...

8. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2152265025017173

AML-732: Ziftomenib Combined With Intensive Induction ... In the ongoing KOMET-007 study, ziftomenib 600 mg QD plus 7+3 was well tolerated with robust clinical activity in newly diagnosed AML. No DS was reported; ...