gilteritinib for Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Site FR33014, Vandoeuvre les Nancy, France
Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation+4 More
gilteritinib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug can prevent relapse in people with acute myeloid leukemia.

See full description

Eligible Conditions

  • Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation
  • Acute Myeloid Leukemia (AML)

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation

Study Objectives

This trial is evaluating whether gilteritinib will improve 2 primary outcomes and 13 secondary outcomes in patients with Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation. Measurement will happen over the course of From date of randomization until the date of documented relapse or discontinuation of the treatment, or initiation of other anti-leukemic treatment or death from any cause; (Median time on study drug was 427 days for gilteritinib and 212 days for Placebo).

Month 3
Change From Baseline in Quantitative Minimal Residual Disease Measured as Log10-transformed Overall FLT3/ITD Mutation Ratio at Months 3, 6, 12, 24/End of Treatment (EoT)
Day 427
Event-Free Survival (EFS)
Day 744
Number of Participants With Adverse Events (AE)
Day 427
Overall Survival (OS)
Day 427
Relapse-free Survival (RFS) Per Independent Review Committee (IRC) Adjudication
Month 1
Number of Participants With Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) Score
Up to 24 months
Eastern Cooperative Oncology Group (ECOG) performance status score
Up to 52 months
Relapse-free Survival (RFS)
Up to 54 months
Minimal Residual Disease (MRD)
Number of participants with abnormal laboratory values and/or adverse events related to treatment
Number of participants with abnormal vital signs and/or adverse events related to treatment
Number of participants with physical exam abnormalities and/or adverse events related to treatment
Safety assessed by Adverse Events (AEs)
Safety assessed by electrocardiograms (ECGs)
Up to 84 months
Event-free survival (EFS)

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation

Trial Design

3 Treatment Groups

ASP2215
1 of 3
Gilteritinib
1 of 3
Placebo
1 of 3
Experimental Treatment
Non-Treatment Group

This trial requires 98 total participants across 3 different treatment groups

This trial involves 3 different treatments. Gilteritinib is the primary treatment being studied. Participants will be divided into 2 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

ASP2215
Drug
Subjects will be treated with ASP2215 once daily (continuously for up to 2 years).
Gilteritinib
Drug
Participants received gilteritinib 120 mg (three tablets of 40 mg) orally, once daily (QD) for up to 2 years or until a protocol-specified discontinuation criterion was met.
Placebo
Drug
Participants received gilteritinib matching placebo orally, QD for up to 2 years or until a protocol-specified discontinuation criterion was met.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gilteritinib
2014
Completed Phase 2
~440
Gilteritinib
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: months 1, 2, 3, 4, 5, 6, 8, 10. 12, 14, 16, 18, 20, 22 and 24/eot
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly months 1, 2, 3, 4, 5, 6, 8, 10. 12, 14, 16, 18, 20, 22 and 24/eot for reporting.

Closest Location

Site US10017 - Gainesville, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received 1 prior treatment for Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation or one of the other 4 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:
Subject is considered an adult according to local regulation at the time of obtaining consent form (ICF).
Subject consents to allow access to subject's diagnostic bone marrow aspirate or peripheral blood sample and/or the DNA derived from that sample, if available, that may be used to validate a companion diagnostic test for gilteritinib.
Subject has confirmed morphologically documented AML, excluding acute promyelocytic leukemia (APL), in CR1 (including CRp and CRi). For the purposes of enrollment, CR will be defined as < 5% blasts in the bone marrow with no morphologic characteristics of acute leukemia (e.g., Auer rods) in the bone marrow with no evidence of extramedullary disease such as central nervous system involvement or granulocytic sarcoma.
Subject will not proceed with transplantation as either a decision not to proceed with transplantation has been made either on the recommendation of the treating physician or by the patient or a suitable donor could not be identified.
Subject is < 2 months from the start of the last cycle of consolidation and should have completed the recommended number of consolidations per local practice.
Subject has had no use of investigational agents, with the exception of FLT3 inhibiting agents during induction and/or consolidation therapy, within the prior 4 weeks.
Subject has had presence of the FLT3/ITD activating mutation in the bone marrow or peripheral blood as determined by the local institution at diagnosis.
Subject has an ECOG performance status 0 to 2.
Serum creatinine ≤ 1.5 x institutional upper limit of normal (ULN), or if serum creatinine outside normal range, then glomerular filtration rate (GFR) > 40 mL/min/1.73m^2 as calculated with the 4-parameter Modification of Diet in Renal Disease (MDRD) equation.
Serum total bilirubin ≤ 2.5 mg/dL (43 μmol/L), except for subjects with Gilbert's syndrome.

Patient Q&A Section

Can leukemia be cured?

"It is well established that chronic myelogenous leukemia can be cured. Therefore our goal should be to not just cure leukemia, but to cure it properly. It is possible that acute myelogenous leukemia can also be cured." - Anonymous Online Contributor

Unverified Answer

What is leukemia?

"Acute myeloid leukemia, an aggressive form of leukemia, is believed to develop when a normal cell is transformed by a mutated gene and, in turn, produces an abnormal accumulation of cells. Acute lymphocytic leukemia is similarly caused by transformation of a normal cell. If leukemia develops in people other than young children, it typically develops in the late part of adulthood." - Anonymous Online Contributor

Unverified Answer

What causes leukemia?

"A combination of factors must be present in the environment for it to become leukemia. A range of environmental exposures can cause leukemia, including chemicals, infections or exposure to radiation. A specific cause is hard to identify, but it is thought to be due to changes in the B cell receptor leading to apoptosis. This has to be tested at the molecular level." - Anonymous Online Contributor

Unverified Answer

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

"Nearly 20.5 million people are impacted by leukemia in the United States each year. This is equal to just over 8% of the United States population. The incidence rate has decreased over the past 20 years, despite the increase in survival. This is most likely due to increased use of therapy and increased diagnostic capabilities to find leukemias with no signs or symptoms." - Anonymous Online Contributor

Unverified Answer

What are common treatments for leukemia?

"The most common treatment for leukemia, according to the analysis of U.S. national data, is chemotherapy, which is an alternative to bone marrow marrow transplantation. A survey of the use of treatments for leukemia in Europe suggests that they are mostly supportive. The authors argue that the use of ancillary drugs, such as anthracyclines and mitoxantrone, in some instances, may be used for the sake of palliation, without considering the role of supportive care, particularly if they are more effective than supportive care." - Anonymous Online Contributor

Unverified Answer

What are the signs of leukemia?

"The following signs and symptoms may be present: fever, malaise, skin changes, chills, weight loss or loss of appetite, blood in stool, and vomiting. All of these sign and symptoms may suggest leukemia. However, there is no single sign or symptom that would allow a doctor to definitively diagnose leukemia. Differentiating between leukemia and other conditions is important, because the signs and symptoms of these diseases are very different." - Anonymous Online Contributor

Unverified Answer

Does leukemia run in families?

"The present study indicates that in contrast to the common and well-known fact that most cases of B-cell chronic lymphocytic leukaemia (CLL) are inherited as an autosomal dominant trait (i.e. MTHFR mutations are seen only in patients with CLL of the familial type), familial cases of CLL are usually monosymptomatic and may arise due to a different inheritance mechanism." - Anonymous Online Contributor

Unverified Answer

How serious can leukemia be?

"Many of the patients with leukemia are not aware of how serious their disease can be. Many doctors don't know what treatment to offer patients. So even while leukemia has a very high (100%) survival rate, some patients still have some pain, and some have to struggle a bit more after the treatment is over." - Anonymous Online Contributor

Unverified Answer

What does gilteritinib usually treat?

"Gilteritinib is a TKI used as an off-label treatment of non-small cell lung cancer (NSCLC), mainly non-squamous and HER1-positive tumors. Given its success in NSCLC, gilteritinib should be considered for the targeted ablation of HER1-positive B-cell leukemias harboring trisomy 12 (myeloid leukemia with trisomy 12). At least some of these patients may benefit from this novel concept. Clinical trials with gilteritinib in B-cell chronic lymphocytic leukemias should be pursued promptly." - Anonymous Online Contributor

Unverified Answer

Has gilteritinib proven to be more effective than a placebo?

"Neither placebo nor the drug met our primary endpoint of ≥20% decrease in BCT, although the drug was significantly more effective than placebo. Data from a recent study demonstrates that gilteritinib has no effect on BCT in patients with MM in clinical practice. We conclude that gilteritinib demonstrates a statistically significant improvement of BCT in patients with MM for which standard therapy with lenalidomide and dexamethasone is available." - Anonymous Online Contributor

Unverified Answer

What are the chances of developing leukemia?

"There are several known and unknown risk factors.\nThere are certain known risk factors as well as genetic factors that increase your risk.\n\n- Smoking: Tobacco smoking is one of the leading causes of leukemia, and the risk is doubled if you smoke for three months or more for twenty years or more.\n- Occupation: Exposure to dust and noise is associated with increased risk.\n- Diet: Certain foods are associated with leukemia.\n- Alcohol: Alcohol has been associated with leukemia in some studies.\n- Exposure to pesticides: Certain pesticides have been associated with leukemia (e.g." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of gilteritinib?

"Side effects of gilteritinib were common and were the same as reported in clinical studies. Median median time to event analysis showed that patients with SLL/CLL experienced a greater median time to first toxicity resolution while PTCLL experienced a greater median time to death. This article is open access under the terms of the Creative Commons Attribution License, where any use of this material by any party requires pre-approval; unless noted otherwise." - 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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