Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)
This research study is for children, teenagers, and young adults whose acute myeloid leukaemia (AML) has returned. It's designed to investigate whether adding a new medication called venetoclax to an existing chemotherapy treatment can improve survival rates. Specifically, it focuses on patients who are experiencing their AML relapse for the second time, or for the first time but cannot receive certain strong chemotherapy drugs. The study aims to understand if venetoclax helps to make the chemotherapy more effective by targeting a specific protein in cancer cells that can make them resistant to treatment. Participants will be randomly assigned to receive either the standard chemotherapy with venetoclax or the standard chemotherapy alone, followed by maintenance treatment.
At a glance
What is this study about?
Acute myeloid leukaemia (AML) is a type of cancer that affects the blood and bone marrow. When AML comes back after initial treatment, it can be more challenging to treat. Some AML cells can become resistant to chemotherapy, making them harder to get rid of. One of the reasons for this resistance is that these cancer cells sometimes produce too much of a protein called BCL-2. This protein acts like a shield, preventing the cancer cells from dying off naturally.
This study is investigating a new drug called venetoclax. Venetoclax works by blocking the BCL-2 protein, which helps to remove the shield from the cancer cells, encouraging them to die. Researchers want to see if adding venetoclax to a standard chemotherapy treatment (a mix of drugs including fludarabine, cytarabine, and gemtuzumab ozogamicin) can improve treatment outcomes for young patients whose AML has relapsed. This is a very important question because AML that has returned often has a less promising outlook.
The study involves children, teenagers, and young adults. It's specifically for those whose AML is coming back for the second time, or for the first time if they cannot have certain other chemotherapy drugs. By comparing treatment with and without venetoclax, the study hopes to determine if this new approach can offer a more effective way to treat these patients and improve their chances of getting better.
Key takeaways
- This study is for young patients (under 21) with AML that has returned.
- It tests if a new drug, venetoclax, improves survival when added to chemotherapy.
- Venetoclax works by helping resistant cancer cells to die.
- Participants are randomly assigned to receive venetoclax or not.
- The goal is to find better treatment options for difficult-to-treat AML.
Who may be eligible?
This study is looking for children, teenagers, and young adults who are at least 29 days old but no older than 21 years of age. They must have acute myeloid leukaemia (AML) that has returned.
Specifically, the AML must be: either a second relapse (meaning it's come back for the second time), or a first relapse (meaning it's come back for the first time) where the doctors believe they can't have certain strong chemotherapy drugs.
Participants also need to be well enough to receive more intensive treatment, as assessed by the medical team. There are also specific timeframes since their last cancer treatment that need to be met, to make sure they've recovered sufficiently.
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 29 days and 21 years old?
- Have you been diagnosed with Acute Myeloid Leukaemia (AML)?
- Has your AML come back (relapsed)?
- Is this your second AML relapse, or your first relapse where you can't have certain other strong drugs?
- Are you generally well enough to start intensive treatment?
What does participation involve?
If you decide to take part in this study, you will be randomly assigned to one of two groups, like flipping a coin. Both groups will receive intensive chemotherapy, but one group will also receive the study drug venetoclax. This treatment will typically last for two cycles, each about 42 days long. During this time, you will have regular medical appointments, blood tests, and other checks to monitor your health and how the treatment is working.
After these initial treatment cycles, if your condition has improved, the aim will be for you to have a stem cell transplant. If a transplant isn't possible, or if the doctors feel you are benefiting, you might continue with a maintenance treatment. This maintenance phase could involve up to 24 cycles (about two years) of treatment with either venetoclax combined with azacitidine, or azacitidine alone, depending on which group you were in. During this time, you would continue to have regular check-ups until the treatment is no longer effective or causes too many side effects.
Potential risks and benefits
Locations (90)
- Phoenix Children's HospitalVerified postcodePhoenix, United States· Recruiting
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States· Recruiting
- MemorialCare Miller Children's and Women's Hospital Long BeachVerified postcodeLong Beach, United States· Recruiting
- Children's Hospital of Orange County Main Campus - OrangeVerified postcodeOrange, United States· Recruiting
- Benioff Children's Hospital - Mission BayVerified postcodeSan Francisco, United States· Recruiting
- Children's Hospital ColoradoVerified postcodeAurora, United States· Recruiting
- Yale UniversityVerified postcodeNew Haven, United States· Recruiting
- Nemours Alfred I. Dupont Hospital for ChildrenVerified postcodeWilmington, United States· Recruiting
- Children's National - Main HospitalVerified postcodeWashington D.C., United States· Recruiting
- Golisano Children's Hospital of Southwest FloridaVerified postcodeFort Myers, United States· Recruiting
- University of Florida Health Shands Children's HospitalVerified postcodeGainesville, United States· Recruiting
- Nemours Children's Specialty Care JacksonvilleVerified postcodeJacksonville, United States· Recruiting
Common questions
What is AML?
AML stands for Acute Myeloid Leukaemia. It's a type of cancer that affects the blood and bone marrow, often needing quick treatment.
What is venetoclax?
Venetoclax is a new medicine being studied. It's designed to help cancer cells die, especially those that have become resistant to other treatments.
Who can join this study?
This study is for children, teenagers, and young adults (from 29 days to 21 years old) whose AML has come back, either for the first time (if other strong drugs aren't suitable) or for the second time.
How long does the treatment last?
The main treatment cycles last about 3 months. If beneficial, a maintenance treatment could then continue for up to 2 years.
Will I definitely get the new drug?
Participants are randomly put into one of two groups. One group gets the new drug (venetoclax) with chemotherapy, and the other gets chemotherapy alone. So, you might or might not receive venetoclax during the initial treatment cycles.
How to find out more
Gwen Nichols, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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