Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML
This research is looking into a new medicine called ziftomenib for people with a blood cancer called Acute Myeloid Leukaemia (AML). Specifically, it's for patients with certain genetic changes in their AML who haven't had treatment for it yet. The study has two parts: one for older or less fit patients, and another for fitter patients. In both parts, ziftomenib will be added to existing standard treatments or a dummy drug (placebo) to see if it improves outcomes. Neither the patient nor their doctor will know if they are receiving ziftomenib or the placebo. The aim is to find out if this new combination is safe and more effective.
At a glance
What is this study about?
This study is a clinical trial, which means it's a carefully planned medical research project. The main goal is to find out if a new drug called ziftomenib can help people with a type of blood cancer called Acute Myeloid Leukaemia (AML). AML is a serious condition where harmful cells grow in the bone marrow, stopping it from making healthy blood cells. Ziftomenib works by targeting a specific pathway in cancer cells, which researchers hope will help fight the disease.
This trial is for adults recently diagnosed with AML who haven't started treatment yet and have specific genetic changes in their cancer (called NPM1-m or KMT2A-r). The study is divided into two main parts. One part is for patients who are older or have other health issues, making them less suitable for very strong treatments. The other part is for patients who are generally fitter and can tolerate more intensive treatments. In both parts, patients will receive their usual AML care, but some will also get ziftomenib, while others will receive a placebo (a dummy drug that looks like ziftomenib).
The reason for using a placebo and not telling anyone who gets which treatment (this is called 'double-blinded') is to make sure the results are as fair and accurate as possible. It helps doctors understand if any changes in patients' health are truly due to ziftomenib rather than other factors. Overall, this study aims to see if adding ziftomenib to current treatments is safe and helps patients with these specific types of AML live longer or have better control over their cancer.
Key takeaways
- This study is testing a new drug, ziftomenib, for a specific type of AML.
- It's for patients who haven't had treatment for AML before and have certain genetic changes.
- You'll receive either ziftomenib or a placebo in combination with standard AML treatment.
- Neither you nor your doctor will know which treatment you are getting.
- The study aims to see if ziftomenib is safe and more effective than current treatments.
- You can stop participating in the study at any time.
Who may be eligible?
To be considered for this study, you need to be at least 18 years old and have been diagnosed with a specific type of AML. This diagnosis must meet the 2022 World Health Organisation (WHO) criteria. You'll also need to be well enough to take part, as assessed by your doctor, and have your liver and kidney functions working adequately.
There are two main groups in the study. If you're 75 or older, or under 75 but with certain health problems that make strong treatments difficult, you might be in the 'Nonintensive Therapy' group. For this group, your AML must have a specific genetic change called NPM1-m. If you're generally fit and able to handle stronger treatments, you might be in the 'Intensive Therapy' group. For this group, your AML should have either the NPM1-m or KMT2A-r genetic change. Your doctor will help decide which group is most suitable for you.
However, there are also reasons why you might not be able to join. For instance, if you've already had treatment for AML (apart from some initial managing treatments), or if you have certain other serious health conditions or types of cancer, you might not be eligible. Your doctor will review all your medical information carefully to see if this study is right 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 at least 18 years old?
- Have you been diagnosed with AML that fits specific criteria?
- Are your AML cells tested for NPM1-m or KMT2A-r genetic changes?
- Have you NOT had previous treatment for your AML (except initial stabilising medicines)?
- Are you generally well enough to participate, as determined by your doctor?
What does participation involve?
If you join this study, you'll be part of a clinical trial that aims to test a new drug called ziftomenib. You'll be assigned to one of two main groups, depending on your overall health and the type of AML you have. Both groups will receive standard AML treatments. On top of this, you’ll either receive ziftomenib or a placebo (a dummy drug). Neither you nor your doctor will know which one you are getting.
The study involves regular visits to the hospital for checks, blood tests, and scans. You'll receive your study medication (ziftomenib or placebo) along with your regular AML treatments. The length of treatment and follow-up will vary depending on your specific study group, but it typically involves an initial treatment phase, followed by further treatment (consolidation), and potentially a longer-term maintenance phase. The total duration of your participation could be several months to a few years, including regular follow-up appointments. You will have regular contact with the study team, and they will monitor your health closely throughout the process. You are free to withdraw from the study at any time.
Potential risks and benefits
Locations (71)
- Banner MD Anderson Cancer CenterVerified postcodeGilbert, United States· Recruiting
- University of California, FresnoVerified postcodeClovis, United States· Recruiting
- University of California, San DiegoVerified postcodeLa Jolla, United States· Recruiting
- Cedars-Sinai Medical CenterVerified postcodeLos Angeles, United States· Recruiting
- University of California, Los AngelesVerified postcodeLos Angeles, United States· Recruiting
- University of California, IrvineVerified postcodeOrange, United States· Recruiting
- University of ColoradoVerified postcodeAurora, United States· Recruiting
- Colorado Blood Cancer InstituteVerified postcodeDenver, United States· Recruiting
- Hartford HealthCare Cancer InstituteVerified postcodeHartford, United States· Recruiting
- Yale University School of MedicineVerified postcodeNew Haven, United States· Recruiting
- University of MiamiVerified postcodeMiami, United States· Recruiting
- Moffitt Cancer Center & Research InstituteVerified postcodeTampa, United States· Recruiting
Common questions
What is AML?
AML stands for Acute Myeloid Leukaemia. It's a type of cancer that starts in the bone marrow, where new blood cells are made, leading to too many abnormal white blood cells.
What is ziftomenib?
Ziftomenib is a new drug being tested. It's designed to target specific pathways in cancer cells, in the hope of stopping them from growing or making them die.
What does 'double-blinded' mean?
It means that neither you nor your doctor will know if you are receiving the new drug (ziftomenib) or a dummy pill (placebo). This helps make the study results fair.
Can I stop being part of the study if I want to?
Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect the medical care you receive afterwards.
Why are there two different groups in the study?
The study has two groups to best suit different patients. One is for those who are older or less fit for very strong treatments, and the other is for fitter patients, each receiving tailored standard care plus the new drug or placebo.
How to find out more
Kura Medical Information
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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