A Phase 1/2, Multicenter, Open-Label, Single Arm, Dose Escalation and Expansion Study of Gilteritinib (ASP2215) Combined with Chemotherapy in Children, Adolescents and Young Adults with FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)
This research study is investigating a new medication called Gilteritinib, given alongside common chemotherapy drugs, for children, teenagers, and young adults. The focus is on those with a specific kind of acute myeloid leukaemia (AML) where the cancer has returned or hasn't improved with earlier treatments. Specifically, it's for AML with a particular genetic change called FLT3/ITD. The study has two main parts: first, finding the safest and most effective dose of Gilteritinib when combined with chemotherapy (Phase 1). Then, in Phase 2, researchers will look at how many patients have their cancer go into remission and how long that remission lasts. The overall goal is to find better ways to treat this challenging form of leukaemia.
At a glance
What is this study about?
This research is looking at a new way to treat a type of blood cancer called acute myeloid leukaemia (AML) in children, teenagers, and young adults. AML is a cancer of the blood and bone marrow. This study focuses on a specific type of AML where the cancer cells have a particular genetic fault called FLT3/ITD. This kind of AML can be harder to treat, especially if it comes back after treatment (relapsed) or doesn't respond to initial treatments (refractory).
The study is testing a new drug called Gilteritinib alongside standard chemotherapy medicines. Gilteritinib works by targeting the specific FLT3/ITD genetic fault in the cancer cells. Researchers want to see if combining Gilteritinib with chemotherapy can be more effective than chemotherapy alone for these patients. The hope is to find a treatment that works better and helps more patients.
The study has two main stages. The first stage (Phase 1) is about finding the right dose of Gilteritinib to use alongside chemotherapy that is both safe and effective. Once the best dose is found, the second stage (Phase 2) will then look at how well this drug combination works. They will measure how many patients' cancer goes into remission, meaning the cancer cells can no longer be found, and how long that remission lasts.
Key takeaways
- This study is for children, teenagers, and young adults with a specific type of AML.
- It's testing a new drug, Gilteritinib, combined with standard chemotherapy.
- The focus is on AML that has returned or not responded to previous treatment.
- The study aims to find the safest and most effective dose, then see how well it works.
- Participation involves regular checks and receiving the study medications.
Who may be eligible?
This study is open to patients who are at least 18 years old, with no upper age limit. Both men and women can take part in this research.
To be eligible, you must have acute myeloid leukaemia (AML) that has a specific genetic change called FLT3/ITD. Your AML must also have either come back after previous treatment (relapsed) or not responded to your previous treatments (refractory).
There will be other detailed medical checks to make sure the study is safe and right for you, which your doctor will discuss.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have Acute Myeloid Leukaemia (AML)?
- Does your AML have the FLT3/ITD genetic change?
- Has your AML come back after treatment or not responded to previous treatment?
What does participation involve?
If you decide to join this study, you'll receive Gilteritinib alongside standard chemotherapy drugs like Zarzio, ARA-cell, Bendarabin, or Citarabina. These medicines will be given as injections or infusions (drips).
The study is divided into two phases. In Phase 1, you'll start with a particular dose of Gilteritinib, and if it's safe, other participants might receive a slightly higher dose until the best balance of safety and effect is found. In Phase 2, everyone will receive this best dose. Throughout the study, you'll have regular hospital visits for blood tests, physical exams, and other checks to see how you're responding to the treatment and to monitor for any side effects. Researchers will also collect samples to check how the drug affects your body and the cancer cells. The total duration of your participation will depend on how you respond to the treatment and the study plan, but it will involve multiple cycles of treatment and follow-up.
Potential risks and benefits
Locations (4)
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedFrance
- —UnverifiedGermany
Common questions
What is AML?
AML stands for Acute Myeloid Leukaemia. It's a type of cancer that starts in the blood-forming cells of the bone marrow.
What does FLT3/ITD positive mean?
This refers to a specific genetic change found in some AML cancer cells. It helps doctors understand more about the type of AML and how it might respond to certain treatments.
What is Gilteritinib?
Gilteritinib is a new drug specifically designed to target the FLT3/ITD genetic change in leukaemia cells. It aims to stop these cells from growing.
What are 'relapsed' or 'refractory' AML?
Relapsed means your AML has come back after treatment. Refractory means your AML didn't respond well to earlier treatments.
Will I get to choose which drugs I receive?
No, in this study, you will receive Gilteritinib combined with specific chemotherapy drugs as part of the study plan. The aim is to test this particular combination.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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