Revumenib for Leukemia

Recruiting at 1 trial location

Overseen By Ghayas Issa, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: M.D. Anderson Cancer Center

Must not be taking: Chemotherapy, Antileukemics

Disqualifiers: Uncontrolled infection, Active malignancy, HIV, 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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if revumenib (also known as Revuforj or SNDX-5613) can control certain types of leukemia linked to increased HOX gene activity. Participants will take revumenib twice daily in 28-day cycles. Suitable candidates include individuals aged 12 and up with a history of leukemia that has recurred or not responded to previous treatments and involves specific genetic changes. The trial excludes those with uncontrolled health issues or active infections. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in leukemia treatment.

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

The trial does not allow the use of other chemotherapeutic or anti-leukemic agents during the study, except for specific cases like intrathecal chemotherapy for CNS leukemia or hydroxyurea for rapidly proliferative disease. If you're on these types of medications, you may need to stop them, but other medications are not specifically mentioned.

Is there any evidence suggesting that revumenib is likely to be safe for humans?

Research has shown that revumenib has been tested for safety in people with leukemia. The FDA has approved revumenib for certain types of leukemia, indicating a known safety profile. However, some serious side effects have been observed. For example, studies have reported that 33% of people experienced febrile neutropenia, a dangerous fever with low white blood cells, and 20% to 29% of people had various infections.

These findings indicate that while revumenib can be effective, it also carries risks. Anyone considering joining a trial should discuss these potential side effects with their doctor to determine if it is a suitable option. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for leukemia, which often involve chemotherapy or targeted therapies like tyrosine kinase inhibitors, Revumenib acts on a novel target. It works by inhibiting the menin-MLL interaction, a mechanism that is crucial in the development of certain types of leukemia. Researchers are excited about Revumenib because it offers a new approach that could potentially be more effective for patients who do not respond well to existing therapies. By specifically targeting this interaction, Revumenib might provide a more precise treatment option with potentially fewer side effects.

What evidence suggests that revumenib might be an effective treatment for leukemia?

Research has shown that revumenib, the treatment under study in this trial, can help treat certain types of leukemia. In one study, 21% of participants experienced complete remission, meaning their leukemia was no longer detectable, or a similar condition called CRh, where some blood cells were still recovering. Another study found that about 23% of patients achieved complete remission or CRh. These patients had tried other treatments before without success. Revumenib targets specific genes linked to leukemia, offering hope for those with difficult cases. ^{1 2 3 6 7}

Who Is on the Research Team?

Ghayas Issa, MD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with leukemia that's linked to high activity of HOX genes. Specific eligibility details are not provided, but typically participants would need a confirmed diagnosis and may have to meet certain health requirements.

Inclusion Criteria

I can take care of myself and am up and about more than half of my waking hours.

My leukemia has returned or is not responding to treatment and has specific genetic changes.

My white blood cell count is below 25,000/uL.

Exclusion Criteria

I currently have an infection that isn't under control.

My leukemia is affecting my brain or spinal cord.

I have severe acute or chronic GVHD.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants take revumenib 2 times a day, every day of each 28-day study cycle

12 months

Monthly 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

• Revumenib

Trial Overview The study is testing the effectiveness of Revumenib, a Menin inhibitor, in managing leukemia where there's an upregulation of HOX genes. It aims to determine if this drug can control the disease.

How Is the Trial Designed?

Group I: Arm 1 Experimental Treatment 1 Intervention

Participants found to be eligible to take part in this study, will take revumenib 2 times a day (each dose about 12 hours apart), every day of each 28-day study cycle.

Revumenib is already approved in United States for the following indications:

Approved in United States as Revuforj for:

• Relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients aged 1 year and older

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

Syndax Pharmaceuticals

Industry Sponsor

Trials

49

Recruited

2,700+

Citations

1. fda.gov fda.gov/drugs/resources-information-approved-drugs/fda-approves-revumenib-relapsed-or-refractory-acute-myeloid-leukemia-susceptible-npm1-mutation

FDA approves revumenib for relapsed or refractory acute ... The main efficacy outcome measures were complete remission rate (CR), CR with partial hematological recovery (CRh), CR+CRh duration, and rate ...

2. ir.syndax.com ir.syndax.com/news-releases/news-release-details/syndax-announces-fda-approval-revuforjr-revumenib-adult-and

Syndax Announces FDA Approval of Revuforj® (revumenib) in ... The rate of complete remission (CR) plus CR with partial hematological recovery (CRh) was 23% (15/65 pts; 95% CI: 14%, 35%). The median time to ...

3. revuforj.com revuforj.com/trial-results/

Trial Results | Revuforj® (revumenib) Official Patient Site In the clinical trial, 21% of people (22 out of 104) achieved complete remission (CR) or complete remission with a partial hematologic recovery (CRh).

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40332046/

Menin inhibition with revumenib for NPM1-mutated ... Revumenib demonstrated clinically meaningful responses in this heavily pretreated, older population with NPM1m AML, including remissions that ...

5. ashpublications.org ashpublications.org/ashclinicalnews/news/8689/Menin-Inhibitor-Revumenib-Yields-Clinically

Menin Inhibitor Revumenib Yields Clinically Meaningful ... In a heavily pretreated population, the CR+CRh rate was 23.4% (n=15) with an ORR of 46.9% (n=30), with 16.7% (n=5) of patients proceeding to ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40437770/

Revumenib for Relapsed or Refractory Acute Leukemia ... Common grade 3/4 adverse reactions reported for revumenib include infectious (febrile neutropenia 33%; infection 29%; bacterial infection 20%) ...

7. ascopubs.org ascopubs.org/doi/10.1200/JCO.24.00826

Menin Inhibition With Revumenib for KMT2A-Rearranged ... Revumenib led to high remission rates with a predictable safety profile in R/R KMT2Ar acute leukemia. To our knowledge, this trial represents ...