Ivosidenib and Azacitidine With or Without Venetoclax in Adult Patients With Newly Diagnosed IDH1-Mutated AML or MDS/AML Considered Ineligible for Intensive Chemotherapy
This study is for adults with a specific type of acute myeloid leukaemia (AML) or MDS/AML that has a particular change in the IDH1 gene. These patients cannot have a strong type of chemotherapy. Currently, the usual treatment combines two drugs: ivosidenib and azacitidine. This trial is investigating whether adding a third drug, venetoclax, to this standard treatment can improve how well patients respond. Researchers want to see if this new combination is safer and more effective. To properly understand the effects of venetoclax, some patients will receive it, while others will receive a 'dummy' drug called a placebo, which has no active ingredients. This helps compare the new treatment fairly and see if venetoclax genuinely makes a difference to patient outcomes.
At a glance
What is this study about?
If you have a type of acute myeloid leukaemia (AML) or MDS/AML with a specific change in your IDH1 gene, and your doctors feel that strong chemotherapy isn't the best option for you, this study might be relevant. The standard treatment for your condition currently involves two medicines: ivosidenib and azacitidine. Doctors are always looking for better ways to treat illnesses, and this study aims to see if adding another drug, called venetoclax, to your current treatment could lead to better results.
The main idea behind this study is to find out if combining these three drugs makes the treatment more effective and safe compared to the standard two-drug approach. To get a clear picture, some patients in the study will receive venetoclax along with ivosidenib and azacitidine, while others will receive a dummy pill (called a placebo) instead of venetoclax. This allows the researchers to fairly compare the two groups and understand if venetoclax truly benefits patients.
Ultimately, this research is important because it could help improve treatment options for people with IDH1-mutated AML or MDS/AML who can't have intensive chemotherapy. The goal is to provide safer and more effective ways to manage this serious condition, helping patients live longer and with a better quality of life.
Key takeaways
- This study evaluates a potentially improved treatment for a specific AML type.
- It's for adults with IDH1-mutated AML who cannot receive strong chemotherapy.
- It combines standard drugs with or without an additional drug, venetoclax.
- A placebo is used to fairly compare the new treatment's effectiveness.
- Participation involves regular hospital visits for treatment and monitoring.
Who may be eligible?
This study is looking for adult patients, aged 18 or over, who have been newly diagnosed with a specific type of acute myeloid leukaemia (AML) or MDS/AML. An important requirement is that your leukaemia must have a change in a gene called IDH1. We will need to confirm this gene change through a special test.
You would be considered for this study if your doctors have decided that strong, intensive chemotherapy is not suitable for you. This decision might be made for several reasons, such as if you are aged 75 or older, or if you are between 18 and 74 but have other health conditions like certain heart or lung problems, or kidney issues, that make intensive chemotherapy too risky. Your doctor will assess your overall health and discuss if you meet these criteria.
Before joining, you will also need to have a healthy enough white blood cell count (which can be managed with medication if needed) and good enough kidney and liver function. Your doctor will carefully review all these points to see if this study is a good fit for you.
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?
- Have you been newly diagnosed with IDH1-mutated AML or MDS/AML?
- Has your doctor said that strong, intensive chemotherapy is not suitable for you?
- Do you have a generally stable health condition, even if you have other illnesses?
- Are you able to have regular hospital visits and follow the study's plan?
What does participation involve?
If you decide to take part in this study, you would receive either the new drug combination (ivosidenib, azacitidine, and venetoclax) or the standard treatment plus a dummy pill (ivosidenib, azacitidine, and a placebo). You won't know which group you are in, but the doctors will. You will have regular hospital visits for check-ups, blood tests, and to receive your treatment. These visits will help the medical team monitor your health, how well the treatment is working, and if you have any side effects.
The drugs will likely be given over a period of time, and the study will involve regular monitoring for as long as you are on treatment. There will also be follow-up appointments even after you stop treatment to see how you are doing in the long term. The exact number of visits and the total length of your participation will be discussed in detail by the study team, as it depends on your individual response and the study's protocol.
Potential risks and benefits
Locations (119)
- AT-Feldkirch-IKHFVerified postcodeFeldkirch, Austria· Not yet recruiting
- AT-Salzburg-SALKVerified postcodeSalzburg, Austria· Not yet recruiting
- AT-Vienna-HANUSCHVerified postcodeVienna, Austria· Not yet recruiting
- BE-Antwerpen-ZASVerified postcodeAntwerp, Belgium· Not yet recruiting
- BE-Brussel-BORDETVerified postcodeBrussels, Belgium· Not yet recruiting
- BE-Brussel-UZBRUSSELVerified postcodeBrussels, Belgium· Not yet recruiting
- BE-Bruxelles-STLUCVerified postcodeBrussels, Belgium· Not yet recruiting
- BE-Gent-UZGENTVerified postcodeGhent, Belgium· Not yet recruiting
- BE-Leuven-UZLEUVENVerified postcodeLeuven, Belgium· Not yet recruiting
- BE-Liege-CHULIEGEVerified postcodeLiège, Belgium· Not yet recruiting
- BE-Yvoir-MONTGODINNEVerified postcodeYvoir, Belgium· Recruiting
- DK-Aalborg-AALBORGUHVerified postcodeAalborg, Denmark· Not yet recruiting
Common questions
What is AML?
AML stands for Acute Myeloid Leukaemia, a type of cancer that starts in the blood-forming cells of the bone marrow.
What does 'IDH1-mutated' mean?
It means the leukaemia cells have a specific change (mutation) in a gene called IDH1. This gene change can affect how the leukaemia behaves.
What is a 'placebo'?
A placebo is a 'dummy' drug or treatment that looks exactly like the real one but has no active ingredients. It helps researchers compare treatments fairly.
Why can't some patients have strong chemotherapy?
Intensive chemotherapy can be very tough on the body. Some patients, especially older ones or those with other health problems, may not be strong enough to safely receive it.
Will I know if I'm getting the new drug or the placebo?
No, you won't know which treatment group you are in. This is called 'blinding' and helps ensure the study results are fair and unbiased.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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