Revumenib Treatment in Combination with FLA Chemotherapy in Paediatric Refractory/Relapsed KMT2A-r, NUP98-r or NPM1-mut Acute Myeloid Leukemia (AML)
This study is looking at a new treatment approach for children and young adults who have Acute Myeloid Leukaemia (AML). This is a type of blood cancer that affects the bone marrow. The study is for patients whose AML has either come back after treatment (relapsed) or hasn't fully responded to previous treatments (refractory). Participants will receive a new drug called Revumenib alongside standard chemotherapy drugs, Fludarabine and Cytarabine. The main goal is to see how many patients respond to this combination treatment. Researchers want to compare the response rate in this study to what has been seen in similar patients in the past, to understand if this new combination is more effective.
At a glance
What is this study about?
This study is about finding better ways to treat Acute Myeloid Leukaemia (AML) in children and young people. AML is a serious type of cancer that starts in the bone marrow, the soft centre of your bones where new blood cells are made. In AML, the body makes too many abnormal white blood cells, which can stop it from making healthy blood cells.
Sometimes, despite treatment, AML can return (this is called 'relapsed') or it might not respond fully to the first treatments (this is called 'refractory'). This study focuses on these difficult-to-treat cases, specifically those with certain genetic changes in their cancer cells (known as KMT2A-r, NUP98-r, or NPM1-mut). These genetic changes are important because they can affect how the cancer behaves and what treatments might work best.
The study is testing a new drug called Revumenib. This drug will be given together with two common chemotherapy drugs, Fludarabine and Cytarabine. The main aim is to see how well this combination treatment works in shrinking the cancer or stopping it from growing. By comparing the results to how patients have responded in previous studies, doctors hope to learn if this new combination could be a more effective treatment option for young people with AML.
Key takeaways
- Targets specific genetic changes in AML (KMT2A-r, NUP98-r, NPM1-mut).
- Tests a new drug (Revumenib) combined with standard chemotherapy.
- For AML that has returned or not responded well to previous treatments.
- Aims to see how many patients respond to this new combination.
- Open to patients aged 18 and older.
Who may be eligible?
This study is for young people and adults who have Acute Myeloid Leukaemia (AML). To join, you must be 18 years old or older. There is no upper age limit for participating.
The study is specifically looking for patients whose AML has either come back after treatment or hasn't fully responded to previous treatments. Your doctors will also check if your AML has specific genetic changes (called KMT2A-r, NUP98-r, or NPM1-mut) as this study is designed for patients with these particular types of AML.
Before joining, you would have a series of tests to make sure that the study treatment is suitable and safe for you, and to confirm that you meet all the necessary health criteria for participation.
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?
- Has your AML come back after treatment, or not improved with previous treatments?
- Have your doctors identified specific genetic changes (like KMT2A-r, NUP98-r, or NPM1-mut) in your AML cells?
- Are you able to attend regular hospital visits for treatment and check-ups?
What does participation involve?
If you decide to take part in this study, you would receive the new drug Revumenib along with standard chemotherapy drugs, Fludarabine and Cytarabine. These treatments would be given in cycles, meaning you would have periods of treatment followed by rest periods.
You would have regular visits to the hospital for check-ups, blood tests, and other assessments to monitor your health, how the treatment is working, and to check for any side effects. These assessments help the doctors understand the treatment's safety and effectiveness.
The total duration of your participation would depend on how your cancer responds to the treatment and your overall health. You would have frequent contact with the study team who would explain everything in detail and support you throughout your time in the study.
Potential risks and benefits
Locations (8)
- —UnverifiedDenmark
- —UnverifiedItaly
- —UnverifiedCzechia
- —UnverifiedSweden
- —UnverifiedPoland
- —UnverifiedNetherlands
- —UnverifiedGermany
- —UnverifiedAustria
Common questions
What is AML?
AML stands for Acute Myeloid Leukaemia, which is a cancer of the blood and bone marrow.
What does 'relapsed' or 'refractory' mean for AML?
'Relapsed' means the cancer has come back after treatment. 'Refractory' means the cancer didn't respond well to initial treatments.
What is Revumenib?
Revumenib is a new drug being tested in this study to treat AML, given alongside standard chemotherapy.
Will I still get normal chemotherapy if I join this study?
Yes, you will receive standard chemotherapy drugs (Fludarabine and Cytarabine) in addition to the new drug, Revumenib.
Who can take part in this study?
This study is for people aged 18 and over, whose AML has relapsed or is refractory, and has specific genetic changes.
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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