A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
This research study, called cAMeLot-1, is testing a new drug called bleximenib for children and adults with acute leukaemia. Acute leukaemia is a type of blood cancer. The main goals are to find the safest and most effective dose of bleximenib (Phase 1) and then to understand how well it works and how safe it is at that dose (Phase 2). This study is for people whose leukaemia has returned or hasn't improved with other treatments. Researchers are particularly interested in leukaemia with specific genetic changes. The study hopes to offer a new treatment option for those who currently have limited choices.
At a glance
What is this study about?
This clinical trial is investigating a new medicine called bleximenib for patients with acute leukaemia. Acute leukaemia is a type of cancer that affects the blood and bone marrow. Sometimes, leukaemia can come back after treatment, or it might not respond to standard medicines, which doctors call 'relapsed or refractory'. For these patients, finding new treatment options is really important.
The study is split into two main parts, called Phase 1 and Phase 2. In Phase 1, the main goal is to carefully find the best dose of bleximenib to use. This involves starting with a low dose and gradually increasing it in different groups of patients, all while keeping a close eye on their safety and any side effects. This helps researchers understand what dose is generally safe and how much the body can tolerate. Once the best dose is found, more people will receive it to confirm its safety.
In Phase 2, the focus shifts to how well bleximenib actually works against the leukaemia at the dose found in Phase 1. Researchers will be looking to see if the medicine can shrink the leukaemia or slow its progression. They will also continue to monitor for any side effects. This study is crucial because it helps scientists learn more about bleximenib and whether it could become a new treatment option for people with specific types of acute leukaemia when other treatments haven't worked.
Key takeaways
- This study is testing a new drug, bleximenib, for acute leukaemia.
- It's for people whose leukaemia has come back or not responded to other treatments.
- Patients need to have specific genetic changes in their leukaemia to be eligible.
- The study aims to find the right dose and see how safe and effective the medicine is.
- Participation involves regular hospital visits, tests, and taking the new medicine.
- You can stop participating at any time if you wish.
Who may be eligible?
This study is looking for both children (aged 2 to under 18 years) and adults (18 years and older) with acute leukaemia. A key requirement is that your leukaemia must have come back after treatment or not responded to other treatments, meaning you've tried other options or can't have them.
Critically, your leukaemia needs to have specific genetic changes which doctors can test for. These changes are often referred to as KMT2A, NPM1, NUP98, or NUP214 alterations. If you're an adult with acute myeloid leukaemia (AML) in Phase 2 of the study, your leukaemia must specifically have KMT2A or NPM1 genetic changes.
Before you can join, doctors will also check your general health, including blood tests for healthy blood cell counts and how well your kidneys are working. Your overall ability to do daily activities (your 'performance status') will also be assessed to make sure you're well enough to take part.
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 between 2 and 17 years old, or 18 years or older?
- Has your acute leukaemia returned or not responded to previous treatments?
- Has your doctor confirmed your leukaemia has certain genetic changes (like KMT2A or NPM1)?
- Are your blood counts and kidney function within acceptable limits?
- Are you generally well enough to take part in a study?
- If you are a woman who could get pregnant, are you willing to use effective birth control during and after the study?
What does participation involve?
Taking part in this study would involve regular hospital visits for check-ups, blood tests, and to receive the study medicine, bleximenib. Doctors and nurses would closely monitor your health, looking for any changes in your leukaemia and any side effects from the medication. You might need to have additional scans or procedures depending on how you respond. The doctors will explain exactly what tests and visits are needed throughout the study.
If you are a woman who could get pregnant, or a man, you would need to agree to use effective birth control during the study and for a period afterwards (90 days for men, 6 months for women) to prevent pregnancy. You would also need to agree not to donate sperm or eggs for future reproductive use during this time. The total duration of your participation would depend on how your leukaemia responds to the treatment and advice from your study doctor.
Potential risks and benefits
Locations (103)
- City of Hope PhoenixVerified postcodeGoodyear, United States· Terminated
- City of HopeVerified postcodeDuarte, United States· Recruiting
- University of California Irvine Medical CenterVerified postcodeOrange, United States· Completed
- University of California San FranciscoVerified postcodeSan Francisco, United States· Recruiting
- UCSF Benioff Children's HospitalVerified postcodeSan Francisco, United States· Recruiting
- University of ChicagoVerified postcodeChicago, United States· Recruiting
- St Francis Hospital & Health Centers Indiana Blood and Marrow TransplantationVerified postcodeIndianapolis, United States· Recruiting
- Norton Cancer InstituteVerified postcodeLouisville, United States· Completed
- Massachusetts General HospitalVerified postcodeBoston, United States· Recruiting
- Dana Farber Cancer InstituteVerified postcodeBoston, United States· Recruiting
- Karmanos Cancer InstituteVerified postcodeDetroit, United States· Recruiting
- Start MidwestVerified postcodeGrand Rapids, United States· Recruiting
Common questions
What is acute leukaemia?
Acute leukaemia is a fast-growing cancer of the blood and bone marrow, which are responsible for making blood cells.
What does 'relapsed or refractory' mean?
It means your leukaemia has either come back after treatment or hasn't responded to previous treatments.
What is bleximenib?
Bleximenib is a new, experimental medicine being tested in this study to see if it can treat acute leukaemia.
Why do you need specific genetic changes?
The researchers believe bleximenib might work best for leukaemia with certain genetic changes, so they are focusing on patients with these specific types.
What happens if I decide to leave the study?
You can leave the study at any time, and it won't affect your regular medical care in any way.
How to find out more
Study Contact
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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