Gilteritinib for Leukemia, Myeloid

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Moffitt Cancer Center, Tampa, FL
Leukemia, Myeloid+3 More
Gilteritinib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a combination of vyxeos and gilteritinib can be used to treat patients with relapsed or refractory FLT3-mutated acute myeloid leukemia.

See full description

Eligible Conditions

  • Leukemia, Myeloid
  • Acute Myeloid Leukemia With FLT3/ITD Mutation

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Leukemia, Myeloid

Study Objectives

This trial is evaluating whether Gilteritinib will improve 1 primary outcome and 3 secondary outcomes in patients with Leukemia, Myeloid. Measurement will happen over the course of Up to 18 months.

Up to 18 months
Complete Remission Rate
Event free survival (EFS)
Maximum Tolerated Dose (MTD)
Up to 5 years
Overall survival (OS)

Trial Safety

Safety Progress

1 of 3

Other trials for Leukemia, Myeloid

Trial Design

2 Treatment Groups

Dose Escalation Arm
1 of 2
Dose Expansion Arm
1 of 2
Experimental Treatment

This trial requires 22 total participants across 2 different treatment groups

This trial involves 2 different treatments. Gilteritinib is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 and are in the first stage of evaluation with people.

Dose Escalation ArmParticipants will receive intravenous Vyxeos on days 1, 3 and 5 and Gilteritinib will be given on days 6-19 of induction therapy. The induction and reinduction dose of Vyxeos is 44mg/m2 daunorubicin and 100mg/m2 of cytarabine with each infusion. Dose level 1: Vyxeos + 120 mg Gilertinib In the event of a dose-limiting toxicity (DLT) at the initial dose level, a dose level minus (-) 1 is permitted Dose Level -1: Vyxeos + 80 mg Gilertinib
Dose Expansion ArmParticipants will receive intravenous Vyxeos on days 1, 3 and 5 and Gilteritinib will be given on days 6-19 of induction therapy in the dose determined in the dose escalation arm.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gilteritinib
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 5 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 5 years for reporting.

Closest Location

Moffitt Cancer Center - Tampa, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Leukemia, Myeloid 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:
Provision of signed and dated informed consent form
Stated willingness to comply with all study procedures and availability for the duration of the study
Eastern Cooperative Oncology Group (ECOG) performance status ≤2
FLT3-ITD or FLT3-TKD mutated AML (non-M3) in 1st or greater relapse or refractory to at least one prior line of AML directed therapy
FLT3 testing must be confirmed at the time of disease relapse
Adequate organ function
Left ventricular ejection fraction (LVEF) ≥50%
Prior anthracycline exposure ≤368 mg/m2 daunorubicin (or equivalent)
Ability to take oral medication and willingness to adhere to the medication regimen
For females of reproductive potential: use of highly effective contraception including double barrier methods (condoms with spermicidal jelly or foam and diaphragm with spermicidal jelly or foam), oral, depo provera, or injectable contraceptives, intrauterine devices and tubal ligation.

Patient Q&A Section

What are the latest developments in gilteritinib for therapeutic use?

"Gilteritinib is an inhibitor of the FGFRs1, 2, 3, 4, 5, 6, 7, 8, 9, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, and 24, which are tyrosine kinase receptors involved in cell proliferation, survival, angiogenesis, migration, invasion, adhesion, and metastasis. These receptors are expressed by several tumor cell types and are implicated in tumor growth and progression. Gilteritinib has been shown to inhibit the growth of cultured human leukemic cells in vitro and xenograft models of human solid tumors in vivo." - Anonymous Online Contributor

Unverified Answer

What is the survival rate for leukemia?

"Survival rate depends on many factors however, it seems that age, gender, and type of leukemia are the strongest predictors of long-term prognosis in patients with acute myelogenous leukemia. Patients with less than 50 years of age have a better prognosis compared to patients over 60 years of age. For acute lymphoblastic leukemia, age was also found to be a predictor of prognosis. Marrow transplantation improves the prognosis tremendously for those with acute leukemia. In 1971, the US National Comprehensive Cancer Network (NCCN) established five-year survival rates for AML, ALL, CLL, and DLBC to be approximately 90%, 80%, 65%, and 40% respectively." - Anonymous Online Contributor

Unverified Answer

What causes leukemia?

"The most common cause of childhood leukemia is exposure to ionizing radiation that occurred during pregnancy, birth, infancy, or adulthood. Certain chemicals also increase the risk, including benzene, cadmium, tobacco smoke and lead. Exposure to certain types of infectious agents such as "Toxoplasma gondii" and human herpesvirus 8 (HHV-8) may also play a role in pediatric leukemia. In children, hereditary syndromes such as Down syndrome, Fanconi anemia, and Wiskott–Aldrich syndrome increase the likelihood of developing childhood leukemia." - Anonymous Online Contributor

Unverified Answer

How serious can leukemia be?

"The majority of patients had been treated previously with conventional chemotherapeutic regimens. Patients with refractory disease were more likely to receive second-line therapy. The survival rate was poor, probably due to advanced age or comorbidities." - Anonymous Online Contributor

Unverified Answer

Does leukemia run in families?

"Findings from a recent study demonstrate significant family aggregation of leukemia risk factors, suggesting that genetic predisposition to leukemia may be inherited genetically in some cases." - Anonymous Online Contributor

Unverified Answer

What is the latest research for leukemia?

"There is huge interest in the new developments in the field of cancer biology, especially concerning the genes in which a mutation is present in a specific part of the genome. These mutations may affect gene expression and thus lead to the development of cancer. Furthermore, they might cause problems during the normal development of an organism. The latter phenomenon is called oncogene, and accounts for many hereditary forms of cancer. Some of the most studied oncogenes include those that code for the products of the MYC (myelocytomatosis related cellular transformation 1), RAS (gene) and BCL2 (B-cell lymphoma 2) protooncogenes." - Anonymous Online Contributor

Unverified Answer

How does gilteritinib work?

"Heclininab has shown promising activity in patients with advanced B-cell malignancies who have received at least two prior lines of therapy. Patients with objective responses had an average progression-free survival of 10.7 months (95% CI, 7.2-14.1) and an overall response rate of 39%. Patients also demonstrated significant reductions in serum lactate dehydrogenase (LDH) levels compared with baseline values, indicating that heclinab may exert its antitumor effects through antiangiogenic mechanisms. However, clinical studies are necessary to confirm these findings. Furthermore, patients with LM/NOS (lymphoma, myeloma, or ALD) should be excluded from this trial." - Anonymous Online Contributor

Unverified Answer

Is gilteritinib typically used in combination with any other treatments?

"Gilteritinib is commonly used in combination with other agents or as monotherapy. Some of its most common combinations are with chemotherapy (e.g., doxorubicin, etoposide), immunotherapy (e.g., pembrolizumab), or targeted therapies (e.g., imatinib, erlotinib, sorafenib). Combinations with immunotherapy and/or targeted therapies are often referred to as "combined immune-targeted therapy" (CIT) and include gilteritinib with pembrolizumab or atezolizumab.Gilteritinib has low toxicity and few side effects compared with many newer drugs." - Anonymous Online Contributor

Unverified Answer

Can leukemia be cured?

"Given the current knowledge of the biology of leukemia, it appears that cure cannot be achieved. This does not mean that patients will die - rather, their chances of survival may be greatly improved by treatment. Despite the overwhelming progress made in gene therapy, the biological basis for chemotherapy remains elusive. However, the problems associated with gene therapy are now being tackled by a new generation of drugs which act directly on the cancerous gene itself and hence have fewer adverse effects. There is also evidence that targeting tumor initiation pathways might provide additional benefits. Current advances in treating CML are directed towards overcoming drug resistance and prolonging survival. In addition, long-term studies of survivors are being conducted at many different institutions around the world." - Anonymous Online Contributor

Unverified Answer

What is gilteritinib?

"Gilteritinib is an oral inhibitor of Src family kinases, including FLT3, PDGFRα, EGFR, and c-Src. Gilteritinib was developed as anticancer agent by GlaxoSmithKline (GSK) based on its role as an inhibitor of the Src tyrosine kinase activity in certain tumor cell lines. In vitro assays demonstrated that gilteritinib has anti-proliferative activity against both normal and malignant cells, and inhibits growth of head and neck squamous carcinoma xenografts grown in athymic mice." - Anonymous Online Contributor

Unverified Answer

How quickly does leukemia spread?

"At present, there is no reliable method for predicting the early spread of leukemias. However, from our experience, we think that a low percentage of patients have advanced stages by the time of diagnosis. For example, our patient population has a median age of 48 years; therefore, he or she may be diagnosed with an indolent disease when the tumor is small enough to remain localized and unnoticed in the bone marrow. And even if the disease did progress to more advanced stages, it progressed slowly because of the low percentage of the patient population who had advanced stages of the disease. We think that this phenomenon might apply to other types of cancers." - Anonymous Online Contributor

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