Gilteritinib for Leukemia

Phase-Based Estimates
1
Effectiveness
2
Safety
Marshfield Medical Center, Marshfield, WI
Leukemia+3 More
Gilteritinib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Leukemia

Study Summary

Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia

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Eligible Conditions

  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Acute Myeloid Leukemia (AML)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Gilteritinib will improve 1 primary outcome and 7 secondary outcomes in patients with Leukemia. Measurement will happen over the course of 3 months.

10 months
Number of participants treatment-related adverse events on Arm A (gilteritinib) as assessed by CTCAE v5.0
Number of participants treatment-related adverse events on Arm B (midostaurin) as assessed by CTCAE v5.0
3 months
CRc (CR or CRi) rate at end of Induction
FLT3 Mutation Negative Composite Complete Response (CRc) [includes Complete Response (CR) or CR with incomplete hematologic recovery (CRi)] at end of Induction
FLT3 mutation negative Complete Response (CR) rate at end of Induction
Minimal Residual Disease (MRD)- CRc rate at end of Induction
68 months
Event Free Survival (EFS)
Overall Survival (OS)

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

2 Treatment Groups

Arm B
Arm A

This trial requires 179 total participants across 2 different treatment groups

This trial involves 2 different treatments. Gilteritinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

Arm AInduction: Daunorubicin, cytarabine and gilteritinib. Depending on response, second cycle of Induction may be given. Consolidation: High-dose cytarabine and gilteritinib.
Arm BInduction: Daunorubicin, cytarabine and midostaurin. Depending on response, second cycle of Induction may be given. Consolidation: High-dose cytarabine and midostaurin.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gilteritinib
FDA approved
Daunorubicin
FDA approved
Cytarabine
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 68 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 68 months for reporting.

Closest Location

Marshfield Medical Center - Marshfield, WI

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Leukemia or one of the other 3 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
People who are having a bone marrow biopsy because they are suspected of having acute myeloid leukemia or who already know they have it will be asked to sign a consent form that allows their samples to be tested at a central lab. show original
The patient must have a form of leukemia that is caused by a change (mutation) in the FLT3 gene, and the leukemia must not have been treated before show original
If Acute Promyelocytic Leukemia (APL) is suspected, hydroxyurea and emergent leukapheresis or preemptive treatment with retinoic acid can be allowed. show original
The patient will start receiving the standard of care chemotherapy using the same regimen and doses as defined in the protocol while awaiting prescreening test results. show original
Patient may not have received the hypomethylating agent within 21 days. show original
It is possible that the patient does not have M3 acute myeloid leukemia. show original
I've undergone therapy in the past for myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPN), including treatments like thalidomide or lenalidomide, interferon, jakafi, cytokines, 5-azacytidine or decitabine, and histone deacetylase inhibitors. show original
The patient may not have AML if they have a known Core Binding Factor -t(8;21), inv(16), or t(16;16) mutation. show original
° If the prescreening test results are not available within 24 hours after randomization, the patient may start standard of care 7+3 chemotherapy using the same regimen and doses as defined in the protocol. show original
The patient may not have been aware that they had active Central Nervous System (CNS) leukemia. show original

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What causes leukemia?

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This disease can be caused by chromosomal abnormalities, inherited mutations in BCR-ABL, and drugs such as arsenic; leukemogenesis is a result of various mechanisms, such as excessive proliferation or an altered metabolic process.

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Can leukemia be cured?

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The majority of adult leukemia is curable. Children and young adults typically have a much poorer prognosis because of their immature cells' inability to mount an immune response against cancer cells. However, the very young child can develop leukemia due to inherited genetic syndromes, and leukemia is one of the most common types of cancer in infants and children. Cancer will be cured for all children (including infants) who are cured of leukemia. Children with ALL and ALL with Down syndrome may be cured by the allogeneic stem cell transplant. Most patients with CLL, younger than age 55, will be found to have some abnormal lymphocytes with mutations in the BCR-ABL fusion gene.

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What is leukemia?

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This disorder is a high risk cancer characterized by the presence of abnormal cells that do not die by apoptosis. Typically it is found in infants, children, or young adults. There are more than 50 types of leukemia. The most common is a form of blood cell cancer that happens in the bone marrow during development.\n

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How many people get leukemia a year in the United States?

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It is estimated that in 2010, approximately 26,900 people will be diagnosed with acute lymphoblastic or acute myeloid leukemia, which accounts for roughly 13% of all new cancer diagnoses in that year. However, it is estimated that only about 2,000 people will die from such leukemia in the year, accounting for less than 1.5% of all cancer-related deaths that year.\n

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What are the signs of leukemia?

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Leukemias are characterized by bleeding, vomiting, swollen lymph nodes, fever, and weight loss. Most of the signs indicate acute leukemia. If there are no symptoms of leukemia, a thorough blood count, differential, and blood film should be performed to determine a diagnosis.\n

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What are common treatments for leukemia?

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Most patients are managed in accordance with established evidence-based treatments, which can be applied in a variety of different ways, depending on the patient and the tumor. In terms of treating ALL, the most common treatment is chemotherapy, which can be given every couple of weeks or every few months to treat leukemia. Patients with CLL or CML receive treatment at a more frequent interval. Oncologists must be vigilant in observing for, and responding appropriately to, toxicity of their disease at any point during their treatment.

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Does leukemia run in families?

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Patients with acute and/or chronic forms of leukemia have a family history of the disease. For some subtypes of acute and/or chronic leukemias, family history is the strongest predictor for disease risk. Patients and their families need to be informed of options for surveillance and prevention.

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Has gilteritinib proven to be more effective than a placebo?

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Gilteritinib has received fast track approval for the treatment of adults with locally advanced or metastatic gastric or gastroesophageal junction adenocarcinomas. We used a prospective blinded clinical trial to test the activity of gilteritinib and as it turned out, gilteritinib has proved its effectiveness against this disease.

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Does gilteritinib improve quality of life for those with leukemia?

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The use of gilteritinib resulted in significant (p<0.0001) improvements in all eight of the components of the EORTC QoL questionnaire as assessed by change in score.

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What is the survival rate for leukemia?

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Patients with high-risk cancer can live a long life, if not with the current treatment. As chemotherapy is the mainstay treatment for chronic leukemia it’d be worthwhile to identify and target patients who are likely to benefit from therapy using only biological agents. It’d be a worthwhile endeavour to find a way to shorten the treatment duration with biological agents, avoiding costly and toxic chemotherapy.

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What is the primary cause of leukemia?

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Lymphocytes are the most commonly mutated cell in [chronic lymphocytic leukemia](https://www.withpower.com/clinical-trials/chronic-lymphocytic-leukemia) and, like acute leukemia, the primary cause of both is mutation of the p53 codon 72. The primary cause of acute myeloid leukemia is mutation of the "NPM1" gene. The primary cause of acute lymphoblastic leukemia is a translocation of chromosome 13, in this case between chromosome 11 and chromosome 21. Recent findings would suggest that, contrary to the current concept, all human leukemias may have the same cause.

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How does gilteritinib work?

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Taken together, these observations indicate that gilteritinib is likely to have significant antitumor activity in patients with JAK2-mutated CRKL-positive or, potentially, other FELIC and/or BCR-ABL-expressing leukemia types. Further development of gilteritinib is warranted.

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