36 Participants Needed

Gilteritinib + Ivosidenib/Enasidenib for Acute Myeloid Leukemia

Recruiting at 6 trial locations
AG
Eytan Stein, MD profile photo
Overseen ByEytan Stein, MD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

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

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

What data supports the effectiveness of the drug Gilteritinib for treating Acute Myeloid Leukemia?

Gilteritinib has been shown to improve survival and response rates in patients with relapsed or refractory acute myeloid leukemia (AML) with FLT3 mutations, compared to standard chemotherapy. It is effective as a single-agent treatment and is well-tolerated, making it a valuable option for these patients.12345

Is the combination of Gilteritinib and Ivosidenib/Enasidenib safe for treating acute myeloid leukemia?

Gilteritinib has been shown to be generally safe in humans, with common side effects including anemia (low red blood cell count), febrile neutropenia (fever with low white blood cell count), and thrombocytopenia (low platelet count). Serious but manageable side effects include differentiation syndrome (a potentially life-threatening condition), QT interval prolongation (heart rhythm changes), and pancreatitis (inflammation of the pancreas). Safety data for Ivosidenib and Enasidenib are not provided in the available research.12346

What makes the drug combination of Gilteritinib, Ivosidenib, and Enasidenib unique for treating acute myeloid leukemia?

This drug combination is unique because it targets specific genetic mutations in acute myeloid leukemia, such as FLT3 and IDH mutations, using oral medications that are more convenient and potentially less toxic than traditional chemotherapy. Gilteritinib is a potent FLT3 inhibitor, while Ivosidenib and Enasidenib target IDH1 and IDH2 mutations, respectively, offering a personalized approach to treatment.12346

What is the purpose of this trial?

The researchers are doing this study to see if the combination of gilteritinib with ivosidenib or enasidenib is a safe and effective treatment for people with relapsed/refractory AML with FLT3/IDH1 or FLT3/IDH2 gene mutations. The researchers will also look for the highest dose of the combination of gilteritinib with ivosidenib or enasidenib that causes few or mild side effects. When the highest safe dose is found, they will test that dose in new groups of participants.

Research Team

Eytan M. Stein, MD - MSK Leukemia ...

Eytan Stein, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Eligibility Criteria

Adults over 18 with relapsed or refractory Acute Myeloid Leukemia (AML) and specific gene mutations (FLT3/IDH1 or FLT3/IDH2). They must be able to follow the study plan, have adequate organ function, and not be pregnant. Men and women must agree to use birth control. Exclusions include uncontrolled infections, severe leukemia complications, heart issues, absorption problems, certain neurological diseases, and those on other cancer trials.

Inclusion Criteria

My tests show FLT3 and IDH mutations in my recent relapse.
My kidney function is good, with a creatinine clearance rate of at least 30 mL/min or my serum creatinine is less than 2.0.
I have had a stem cell transplant for my AML.
See 8 more

Exclusion Criteria

I have a history of a brain infection called PML.
I have an ongoing serious infection that isn't under control.
Patient on any other investigational anti-cancer agents
See 9 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the combination of gilteritinib with ivosidenib or enasidenib in continuous 28-day cycles

1 year
Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Enasidenib
  • Gilteritinib
  • Ivosidenib
Trial Overview The trial is testing if combining gilteritinib with ivosidenib or enasidenib is safe and effective for AML patients with certain genetic mutations. It aims to find the highest dose that causes few side effects before using this dose in new participant groups.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: gilteritinib + ivosidenib (Cohort 1)Experimental Treatment2 Interventions
Each patient will take the combination of gilteritinib/ ivosidenib (Cohort 1) , daily, in continuous 28-day cycles at the dose level that they are assigned.
Group II: gilteritinib + enasidenib (Cohort 2)Experimental Treatment2 Interventions
Each patient will take the combination of gilteritinib/enasidenib (Cohort 2) daily, in continuous 28-day cycles at the dose level that they are assigned.

Enasidenib is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Idhifa for:
  • Relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation
🇪🇺
Approved in European Union as Idhifa for:
  • Acute myeloid leukaemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation

Find a Clinic Near You

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials
1,998
Recruited
602,000+

Astellas Pharma US, Inc.

Industry Sponsor

Trials
90
Recruited
12,900+

Naoki Okamura

Astellas Pharma US, Inc.

Chief Executive Officer since 2023

Not available

Tadaaki Taniguchi

Astellas Pharma US, Inc.

Chief Medical Officer since 2023

MD, PhD

Findings from Research

In a phase 3 trial involving 371 patients with relapsed or refractory FLT3-mutated acute myeloid leukemia (AML), gilteritinib significantly improved overall survival compared to salvage chemotherapy, with a median survival of 9.3 months versus 5.6 months.
Gilteritinib also led to higher rates of complete remission (21.1% vs. 10.5%) and had fewer severe adverse events compared to chemotherapy, indicating it is a safer and more effective treatment option for this patient population.
Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML.Perl, AE., Martinelli, G., Cortes, JE., et al.[2023]
Gilteritinib fumarate is an effective and well-tolerated treatment for patients with relapsing or refractory FLT3-positive acute myeloid leukemia, showing promise in managing the disease with lower toxicity compared to earlier treatments.
The drug's oral formulation makes it suitable for a wide range of patients, including elderly and frail individuals, allowing for outpatient treatment that reduces hospitalization risks and costs.
Therapeutic Management of Patients with FLT3 + Acute Myeloid Leukemia: Case Reports and Focus on Gilteritinib Monotherapy.Bocchia, M., Carella, AM., Mulè, A., et al.[2022]
Gilteritinib significantly improves overall survival and response rates in adults with relapsed or refractory acute myeloid leukaemia (AML) with FLT3 mutations, as demonstrated in the phase III ADMIRAL trial.
While gilteritinib is effective, it can cause serious side effects like anaemia and febrile neutropenia, but these are generally manageable with dose adjustments, highlighting the importance of monitoring during treatment.
Gilteritinib: A Review in Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukaemia.Kang, C., Blair, HA.[2021]

References

Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML. [2023]
Therapeutic Management of Patients with FLT3 + Acute Myeloid Leukemia: Case Reports and Focus on Gilteritinib Monotherapy. [2022]
Gilteritinib: A Review in Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukaemia. [2021]
Gilteritinib in Combination With Induction and Consolidation Chemotherapy and as Maintenance Therapy: A Phase IB Study in Patients With Newly Diagnosed AML. [2023]
[Pharmacological and clinical profile of gilteritinib (Xospata® tablets 40 mg), a therapeutic agent for relapsed or refractory FLT3-mutated acute myeloid leukemia]. [2021]
FDA Approval Summary: Gilteritinib for Relapsed or Refractory Acute Myeloid Leukemia with a FLT3 Mutation. [2023]
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