Study of Revumenib in Combination With Intensive Chemotherapy in Newly Diagnosed Acute Myeloid Leukemia (AML) With a NPM1 Mutation
This study is investigating a new medication called revumenib. It's being tested in people who have recently been diagnosed with a type of blood cancer called acute myeloid leukaemia (AML), specifically those with a gene change known as an NPM1 mutation. The main goal is to find out if adding revumenib to the usual chemotherapy treatment can improve how well people do, compared to having chemotherapy alone. Researchers will also be carefully checking for any side effects and how safe this new combination treatment is. This is a Phase 3 study, which means it’s one of the final steps before a new medicine might become widely available, if proven safe and effective.
At a glance
What is this study about?
This research study is focused on a specific type of blood cancer called Acute Myeloid Leukaemia (AML). When someone is newly diagnosed with AML, doctors try to find the best possible treatment. This study is exploring a new medicine, revumenib, and trying to see if it can improve how well treatments work when given alongside standard chemotherapy. The researchers are particularly interested in people whose AML has a specific change in their genes, called an NPM1 mutation. This is important because understanding these changes can help doctors tailor treatments more effectively.
The study aims to compare two groups of patients. One group will receive the standard chemotherapy, which is the current best treatment. The other group will receive the same standard chemotherapy, but with the addition of revumenib. By comparing these groups, the researchers hope to determine if adding revumenib leads to better results for patients, such as longer periods of remission or improved overall health outcomes. They will also be closely monitoring the safety of this combination treatment to understand any potential side effects.
Ultimately, the goal of this study is to find new and more effective ways to treat AML, especially for those with the NPM1 mutation. If the study shows that revumenib is both safe and effective when combined with chemotherapy, it could potentially change how this type of AML is treated in the future, offering new hope for patients.
Key takeaways
- This study evaluates a new drug, revumenib, for newly diagnosed AML.
- It targets AML patients with a specific gene change (NPM1 mutation).
- Revumenib is given in combination with standard chemotherapy.
- The study aims to see if this combination improves outcomes and is safe.
- Participation involves close monitoring and regular clinic visits.
- Patients may receive revumenib or a dummy pill (placebo) with chemotherapy.
Who may be eligible?
To be considered for this study, you would need to be an adult or young person, at least 12 years old, who has recently been diagnosed with Acute Myeloid Leukaemia (AML) and hasn't started treatment yet. A key requirement is that your AML must have a specific gene change called an 'NPM1 mutation'. You'd also need to be generally well enough to manage intensive chemotherapy, which your doctor would assess.
There are also some health conditions that would prevent someone from joining the study. For example, if you have another type of leukaemia called active acute promyelocytic leukaemia, or if the cancer has spread to your brain or spinal cord. Certain heart conditions, especially those affecting your heart's electrical activity (like a long QT interval), would also mean you couldn't take part, as would some serious digestive problems that might stop the oral medication from being properly absorbed.
Additionally, if you are pregnant, breastfeeding, or have certain active infections like hepatitis B, active hepatitis C, or uncontrolled HIV, you wouldn't be able to join. Your doctors would perform several tests to ensure you meet all the health requirements for the study, including checking your organ function and overall health status.
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.
- Have you been newly diagnosed with AML?
- Does your AML have an NPM1 gene change?
- Are you generally well enough for intensive chemotherapy?
- Are you at least 12 years old?
- Are you male, or a female who is not pregnant or breastfeeding?
- Do you have good kidney, liver, and heart function?
What does participation involve?
If you decide to take part in this study, you would receive either the new medicine (revumenib) plus standard chemotherapy, or standard chemotherapy plus a dummy pill (placebo). You wouldn't know which group you are in. Your medical team would closely monitor your health throughout the treatment period. This would involve regular visits to the clinic for check-ups, blood tests, and other assessments to see how you are responding to treatment and to check for any side effects. You would continue to take the study medication as prescribed, likely orally, for a set period. After the main treatment, there would be follow-up appointments to track your long-term health and any ongoing progress. The total duration of your participation, including follow-up, would be explained in more detail by the study team.
Potential risks and benefits
Locations (24)
- Clinical Trial SiteVerified postcodeMelbourne, Australia· Recruiting
- Clinical Trial SiteVerified postcodeGosford, Australia· Recruiting
- Clinical Trial SiteVerified postcodeBatumi, Georgia· Recruiting
- Clinical Trial SiteVerified postcodeTbilisi, Georgia· Recruiting
- Clinical Trial Site 1Verified postcodeTbilisi, Georgia· Recruiting
- Clinical Trial Site 2Verified postcodeTbilisi, Georgia· Recruiting
- Clinical Trial SiteVerified postcodeTbilisi, Georgia· Recruiting
- Clinical Trial SiteVerified postcodeDüsseldorf, Germany· Recruiting
- Clinical Trial SiteVerified postcodeHerne, Germany· Recruiting
- Clinical Trial SiteVerified postcodeJena, Germany· Recruiting
- Clinical Trial SiteVerified postcodeJerusalem, Israel· Recruiting
- Clinical Trial SiteVerified postcodeCandiolo, Italy· Recruiting
Common questions
What is AML?
AML stands for Acute Myeloid Leukaemia. It's a fast-growing cancer of the blood and bone marrow, which are responsible for making blood cells.
What is an NPM1 mutation?
An NPM1 mutation is a specific change found in the genes of some people with AML. It helps doctors understand more about the specific type of AML a person has.
What does 'Phase 3' mean for a study?
Phase 3 is usually one of the final steps in testing a new medicine. It means the treatment has already shown some promise and is now being compared to existing treatments in a larger group of people to confirm its effectiveness and safety.
Will I know if I'm getting the new drug or the placebo?
In this study, you will not know whether you are receiving revumenib or a placebo (dummy pill) alongside your standard chemotherapy. This is a common practice in clinical trials to ensure the results are unbiased.
Can I stop participating if I want to?
Yes, you are completely free to withdraw from the study at any time, for any reason, without it affecting your medical care.
How to find out more
Syndax Pharmaceuticals
Always speak to your GP or specialist before deciding to take part in a study.
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