#1 Clinical Trials Search in Europe

Gilteritinib

Gilteritinib is an emerging drug being studied in clinical trials for the treatment of acute myeloid leukemia (AML), particularly in patients with FLT3 gene mutations. This article explores the use of gilteritinib in various clinical trials, examining its efficacy, safety, and potential benefits for AML patients. We’ll look at how gilteritinib is being tested in different patient populations and treatment settings to better understand its role in improving outcomes for those with this challenging form of leukemia.

Table of Contents

What is Gilteritinib?

Gilteritinib is a medication used in the treatment of certain types of blood cancers. It is also known by its brand name Xospata® and its research code ASP2215[7]. Gilteritinib belongs to a class of drugs called tyrosine kinase inhibitors, which work by targeting specific proteins in cancer cells to slow down or stop their growth[2].

How Does Gilteritinib Work?

Gilteritinib works by specifically targeting and inhibiting two proteins called FLT3 and AXL. These proteins are often overactive in certain types of leukemia cells, causing them to grow and divide uncontrollably. By blocking these proteins, gilteritinib can help stop the growth of cancer cells and potentially lead to their death[3].

The drug is particularly effective against leukemia cells that have mutations in the FLT3 gene. These mutations can make the cancer more aggressive and harder to treat with standard therapies. Gilteritinib is designed to work against both common types of FLT3 mutations: FLT3-ITD and FLT3-TKD[3].

What Conditions Does Gilteritinib Treat?

Gilteritinib is primarily used to treat a specific type of blood cancer called Acute Myeloid Leukemia (AML). AML is a cancer of the blood and bone marrow that progresses rapidly without treatment. Specifically, gilteritinib is used in the following situations:

  • Relapsed or Refractory AML: This means the cancer has either come back after initial treatment (relapsed) or did not respond well to previous treatments (refractory)[4].
  • FLT3-positive AML: This refers to AML that has mutations in the FLT3 gene, which gilteritinib specifically targets[5].
  • Newly Diagnosed AML: Some clinical trials are exploring the use of gilteritinib in combination with other drugs for patients who have just been diagnosed with FLT3-positive AML[3].

How is Gilteritinib Administered?

Gilteritinib is taken orally, usually once daily. The typical dose is 120 mg per day, but this can vary depending on individual patient factors and how well the drug is tolerated[1]. The medication is often given in 28-day cycles, and treatment may continue as long as the patient is benefiting from it and not experiencing severe side effects.

It’s important to take gilteritinib exactly as prescribed by your doctor. The drug should be taken at least 2 hours after or 1 hour before food[2]. Your doctor will monitor your response to the treatment and may adjust the dose if necessary.

Gilteritinib in Clinical Trials

Gilteritinib has been and continues to be studied in various clinical trials to better understand its effectiveness, safety, and potential new uses. Some key areas of research include:

  • Combination Therapy: Studies are exploring the use of gilteritinib in combination with other drugs, such as venetoclax and azacitidine, to potentially improve outcomes for patients with FLT3-mutated AML[5].
  • Newly Diagnosed AML: While gilteritinib is approved for relapsed/refractory AML, trials are investigating its use as part of initial treatment for newly diagnosed patients[3].
  • Different Formulations: Researchers are studying various forms of gilteritinib, including tablets and oral suspensions, to determine the best ways to administer the drug[2].
  • Long-term Effects: Ongoing studies are monitoring patients who have been treated with gilteritinib to understand its long-term efficacy and safety profile[6].

Potential Side Effects and Safety Considerations

Like all medications, gilteritinib can cause side effects. Common side effects may include:

  • Fatigue
  • Nausea and vomiting
  • Diarrhea
  • Decreased appetite
  • Changes in liver function tests
  • Low blood cell counts (which can increase the risk of infections, anemia, and bleeding)

More serious side effects can occur, including a condition called differentiation syndrome, which can cause fever, difficulty breathing, and other symptoms. It’s crucial to report any new or worsening symptoms to your healthcare provider promptly[7].

Your doctor will monitor you closely during treatment, which may include regular blood tests and other assessments to check for side effects and evaluate how well the treatment is working[8].

Gilteritinib in Special Populations

Research is ongoing to understand how gilteritinib affects different groups of patients:

  • Patients with Kidney Problems: Studies have looked at how severe kidney impairment affects the way gilteritinib is processed in the body[7].
  • Patients with Liver Problems: Research has also been conducted to understand how liver impairment impacts gilteritinib’s effects and safety[8].
  • Older Adults: As AML can affect older individuals, studies are examining how age might influence the effectiveness and safety of gilteritinib[9].

It’s important to discuss your full medical history with your doctor before starting gilteritinib treatment to ensure it’s the right option for you.

Aspect Details
Drug Name Gilteritinib (ASP2215, XOSPATA®)
Primary Use Treatment of Acute Myeloid Leukemia (AML), especially in patients with FLT3 mutations
Administration Oral tablet, typically once daily
Key Clinical Trials ADMIRAL, COMMODORE, and various Phase 1/2 studies
Patient Populations Newly diagnosed AML, relapsed/refractory AML, post-stem cell transplant
Efficacy Measures Complete remission rates, overall survival, event-free survival, relapse-free survival
Safety Assessments Adverse events, laboratory evaluations, ECG, vital signs
Combination Therapies Being studied with venetoclax, azacitidine, and as part of triple regimens
Special Populations Studies in patients with renal impairment and hepatic impairment
Ongoing Research Optimal dosing, long-term efficacy, use in various AML subtypes and treatment settings

FAQ

  • What is gilteritinib? Gilteritinib is a targeted therapy drug used to treat acute myeloid leukemia (AML) in patients with a specific genetic mutation called FLT3. It works by blocking the activity of the mutated FLT3 protein, which can help slow or stop the growth of leukemia cells.
  • How is gilteritinib administered? Gilteritinib is typically taken orally as a tablet once daily. In clinical trials, it has been administered in various doses, ranging from 20 mg to 200 mg, depending on the study and patient needs.
  • What types of patients are being studied in gilteritinib clinical trials? Clinical trials are studying gilteritinib in various patient groups, including those with newly diagnosed AML, relapsed or refractory AML, and patients who have undergone stem cell transplantation. Some studies focus specifically on patients with FLT3 mutations.
  • What are some potential side effects of gilteritinib? While side effects can vary, some common ones observed in clinical trials include changes in liver function, diarrhea, fatigue, and changes in blood cell counts. The safety profile is being closely monitored in ongoing studies.
  • How does gilteritinib compare to other AML treatments? Some clinical trials are comparing gilteritinib to standard chemotherapy or other targeted therapies. Early results suggest it may be effective in certain patient groups, particularly those with FLT3 mutations, but more research is needed to fully understand its comparative efficacy.

Ongoing Clinical Trials on Gilteritinib

  • Study on the Effectiveness of Gilteritinib and Drug Combination for Patients with Relapsed or Refractory Acute Myeloid Leukemia

    Recruiting

    1 1 1 1
    Investigated drugs:
    Czechia Germany Italy Lithuania Portugal Romania +1

  • Study of Gilteritinib, Venetoclax, and Azacitidine for Patients with Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutations Not Eligible for Intensive Treatment

    Recruiting

    1 1 1
    Investigated drugs:
    Germany

  • A Study of Gilteritinib to Eliminate Remaining Cancer Cells in Patients with Acute Myeloid Leukemia and FLT3-ITD Mutation

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Greece

  • Study of gilteritinib with fludarabine, cytarabine and idarubicin combination therapy in newly diagnosed FLT3-positive acute myeloid leukemia patients

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

  • Study Comparing Gilteritinib and Midostaurin with Chemotherapy for Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome with FLT3 Mutation

    Not recruiting

    1 1 1
    Investigated drugs:
    Austria Belgium Finland France Germany Ireland +5

  • Study on Gemtuzumab Ozogamicin and Gilteritinib for Adults with Relapsed or Refractory Acute Myeloid Leukemia with FLT3 Mutation

    Not recruiting

    1 1 1
    Investigated drugs:
    France

  • Study of Gilteritinib and Chemotherapy for Children, Adolescents, and Young Adults with Relapsed or Refractory Acute Myeloid Leukemia with FLT3 Mutation

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Italy Spain

  • Study of Gilteritinib and Azacitidine for Newly Diagnosed Acute Myeloid Leukemia with FLT3 Mutation in Patients Not Eligible for Intensive Chemotherapy

    Not recruiting

    1 1 1 1
    Investigated drugs:
    Belgium Germany

  • Study on Azacitidine and Gilteritinib for Adults with Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France

Glossary

  • Acute Myeloid Leukemia (AML): A type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells that interfere with the production of normal blood cells.
  • FLT3 mutation: A genetic change in the FLT3 gene that can occur in some AML patients, often associated with a more aggressive form of the disease and poorer prognosis.
  • Relapsed/Refractory AML: AML that has returned after initial treatment (relapsed) or has not responded to treatment (refractory).
  • Hematopoietic Stem Cell Transplantation (HSCT): A procedure in which healthy blood stem cells are transplanted into a patient to replace diseased or damaged bone marrow.
  • Bioavailability: The extent and rate at which a drug enters the body's circulation and becomes available at the site of action.
  • Pharmacokinetics: The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Complete Remission (CR): A state in which there is no evidence of disease and blood counts have returned to normal levels.
  • Measurable Residual Disease (MRD): The small number of cancer cells that may remain in the body during or after treatment, often undetectable by standard tests.
  • Event-Free Survival (EFS): The length of time after treatment during which no specified events (such as disease progression or relapse) are detected.
  • Overall Survival (OS): The length of time from either the date of diagnosis or the start of treatment that patients are still alive.

References

  1. https://clinicaltrials.gov/study/NCT03315299
  2. https://clinicaltrials.gov/study/NCT03964038
  3. https://clinicaltrials.eu/trial/study-of-gilteritinib-with-fludarabine-cytarabine-and-idarubicin-combination-therapy-in-newly-diagnosed-flt3-positive-acute-myeloid-leukemia-patients/
  4. https://clinicaltrials.gov/study/NCT02181660
  5. https://clinicaltrials.gov/study/NCT06561880
  6. https://clinicaltrials.gov/study/NCT02561455
  7. https://clinicaltrials.gov/study/NCT04699877
  8. https://clinicaltrials.gov/study/NCT02571816
  9. https://clinicaltrials.gov/study/NCT05791890