Active not recruitingPHASE1, PHASE2INTERVENTIONAL

First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

This study is looking at a new drug called ziftomenib for people with acute myeloid leukaemia (AML) that has returned or didn't get better with other treatments. It’s also being tested in some people with acute lymphoblastic leukaemia (ALL). Researchers want to see if the drug is safe and how well it works. They are also checking how it interacts with other common medicines. The study has several parts, including an initial phase to find the right dose and then further stages to learn more about its effects. Currently, the main AML study is full for new participants, but other parts are still ongoing.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Kura Oncology, Inc.

Enrolment target

263

Start

12 Sep 2019

Estimated completion

16 Oct 2028

Advanced Malignant Neoplasm Acute Myeloid Leukemia Mixed Lineage Leukemia Mixed Lineage Acute Leukemia Acute Leukemia of Ambiguous Lineage Mixed Phenotype Acute Leukemia Acute Lymphoblastic Leukemia

What is this study about?

This research study is about a new drug called ziftomenib. It's designed to treat certain types of blood cancer, primarily acute myeloid leukaemia (AML) that has either returned after treatment or didn't get better from other therapies. This is a very important step because it’s the very first time this drug is being given to people, so the researchers are carefully checking to make sure it's safe and to see what effects it has.

The study has different parts. The main part, which has finished taking new people, focused on finding the right dose of ziftomenib for AML and seeing how safe it is. There are also four smaller studies, called sub-studies, looking at different things. For example, some sub-studies are checking how ziftomenib works in people with AML who have specific genetic changes. Others are observing how ziftomenib interacts with other common medicines and whether it might help people with a similar type of blood cancer called acute lymphoblastic leukaemia (ALL) that has also come back or not responded to other treatments.

By doing these studies, doctors hope to learn if ziftomenib can be a helpful new treatment option for people with these serious blood cancers, especially those for whom other treatments haven't worked well. They are carefully observing how the drug affects people's bodies and how it might help fight the cancer.

Key takeaways

Tests a new drug, ziftomenib, for specific blood cancers.
Primarily for relapsed or difficult-to-treat AML.
First time the drug is given to people, focusing on safety.
Eligibility often requires specific genetic changes in the cancer.
Some parts of the study are still open for new participants.
Regular hospital visits and health monitoring are needed.

Who may be eligible?

This study is generally for adults aged 18 or over who have acute myeloid leukaemia (AML) that has come back or hasn't responded to previous treatments. This includes people who have already tried standard treatments like a stem cell transplant, or are unable to have them. Importantly, your AML needs to have specific genetic changes, such as a 'KMT2A-r' or 'NPM1-m' mutation, depending on which part of the study you might join. Some parts of the study are also open to people with a similar cancer called acute lymphoblastic leukaemia (ALL) with the 'KMT2A-r' genetic change.

To be considered, you should generally be well enough to take part in a study, meaning you can mostly look after yourself. Your liver and kidney functions need to be healthy, and your blood counts, specifically your white blood cell count, should be within certain limits. If you are a woman who could get pregnant, or a man with a female partner who could get pregnant, you would need to use effective contraception during and for a period after the study.

You would not be able to join if you have certain other medical conditions, such as a specific type of leukaemia called acute promyelocytic leukaemia, or if you have leukaemia that has spread to your brain or spinal cord. Also, if you’ve recently had a stem cell transplant or are receiving certain immune-suppressing medicines, this study might not be right for you. Your medical team will carefully check all these details to see if this study could be an option.

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?
Do you have AML that has come back or not responded to previous treatments, or specific types of ALL?
Does your cancer have specific genetic changes (your doctor can check for KMT2A-r, NPM1-m, or others)?
Are you generally well enough to manage most daily activities?
Do you have healthy liver and kidney function?
Are you willing to use effective contraception if you or your partner can become pregnant?

Answer every question to see your result.

What does participation involve?

If you take part in this study, you will be given the study drug, ziftomenib, likely as tablets. You would also have regular visits to the clinic for check-ups. During these visits, doctors and nurses will monitor your health, take blood and other samples, and ask you how you are feeling. This helps them understand how the drug is affecting your body and the cancer. For some parts of the study, you might also be given other common medicines like Midazolam or Itraconazole to see how they interact with ziftomenib. The total length of time you would be in the study can vary, but typically involves ongoing treatment and monitoring as long as the drug is helping and you are tolerating it well. There will also be a follow-up period after you stop taking the study drug.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as getting access to a new drug that isn't yet widely available, which might help your condition when other treatments haven't. However, there are also potential risks as ziftomenib is a new drug, and not all its effects are known. You might experience side effects, some of which could be serious. The research team will explain all known and possible side effects to you. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual care.

Locations (43)

Banner MD Anderson Cancer Center
Verified postcode
Gilbert, United States
UCLA Ronald Reagan Medical Center
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Los Angeles, United States
Mayo Clinic
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Jacksonville, United States
Robert H. Lurie Comprehensive Cancer Center of Northwestern University
Verified postcode
Chicago, United States
University of Maryland Greenebaum Comprehensive Cancer Center
Verified postcode
Baltimore, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
University of Michigan Hospitals
Verified postcode
Ann Arbor, United States
Karmanos Cancer Institute
Verified postcode
Detroit, United States
Mayo Clinic
Verified postcode
Rochester, United States
Hackensack University Medical Center - John Theurer Cancer Center
Verified postcode
Hackensack, United States
Roswell Park Comprehensive Cancer Center
Verified postcode
Buffalo, United States
Weill Cornell Medical College - NY Presbyterian Hospital
Verified postcode
New York, United States

Common questions

What is ziftomenib?

Ziftomenib is a new drug being tested for blood cancers like AML and ALL. It works differently from other treatments by targeting specific pathways in cancer cells.

What types of cancer is this study looking at?

Mainly acute myeloid leukaemia (AML) that has come back or not responded to previous treatments. It's also being studied for some cases of acute lymphoblastic leukaemia (ALL).

Are there specific genetic changes needed to join?

Yes, for many parts of the study, your cancer needs to have certain genetic changes like KMT2A-r or NPM1-m mutations.

What does 'first in human' mean?

It means this is the first time the drug ziftomenib is being given to people, so researchers are carefully watching for safety and to see what effects it has.

Can I still join the study?

The main part of the study for AML is now full. However, some of the smaller sub-studies are still looking for participants with specific genetic changes or ALL. Your doctor can check if you might be eligible for these.