Active not recruitingPHASE1INTERVENTIONAL

A Study of Bleximenib in Combination With Acute Myeloid Leukemia (AML) Directed Therapies

This research is looking at a new medicine called Bleximenib. It's being tested with existing treatments for a type of blood cancer called acute myeloid leukaemia (AML). The main goals are to figure out the best and safest dose of Bleximenib to use when combined with other AML drugs, and to see what side effects people might experience. This is an early-stage study, meaning it's one of the first times Bleximenib is being given to people in this way. It includes both adults and teenagers with AML that has either come back or hasn't responded to previous treatments.

At a glance

Status

Active not recruiting

Phase

PHASE1

Sponsor

Janssen Research & Development, LLC

Enrolment target

196

Start

04 Oct 2022

Estimated completion

03 Mar 2027

Leukemia, Myeloid, Acute

What is this study about?

This study is investigating a new medicine called Bleximenib for people with acute myeloid leukaemia (AML). AML is a type of cancer that affects the blood and bone marrow, and it needs quick and effective treatment. In this study, researchers want to see how Bleximenib works when given together with other standard AML treatments like Venetoclax, Azacitidine, Cytarabine, Daunorubicin, or Idarubicin. The aim is to find out the best amount, or dose, of Bleximenib to use so it's both safe and effective.

This is a 'Phase 1' study, which means it's one of the first human trials. The main focus at this stage is usually to check the safety of a new drug and find the right dose. After finding a suitable dose, the study will expand to gather more safety information. It's an important step in developing new ways to fight AML.

The study is particularly looking at people whose AML has either returned after treatment (relapsed) or hasn't responded to previous treatments (refractory). This research could help to improve future treatment options for patients with AML, especially for those cases that are harder to treat.

Key takeaways

This study tests a new drug, Bleximenib, for acute myeloid leukaemia (AML).
It aims to find the safest and most effective dose of Bleximenib with other AML treatments.
Teenagers (12+) with relapsed/refractory AML can participate.
This is an early-stage (Phase 1) study focused on safety and dosing.
Participants will receive Bleximenib plus standard AML therapies.

Who may be eligible?

To join this study, participants must be at least 12 years old. Teenagers between 12 and 18 years old can only join if their AML has come back or hasn't responded to previous treatments. All participants must have received a clear diagnosis of AML and their cancer cells must show specific genetic changes (called KMT2A, NPM1, NUP98, or NUP214 alterations).

You would also need to have generally good health measurements like white blood cell count, and your liver and kidney functions should be working well. Adults need to have a certain level of physical ability, while teenagers have specific scales (Lansky or Karnofsky) to check their ability to perform daily activities. Women who could become pregnant would need to have a negative pregnancy test before starting.

However, you wouldn't be able to join if you have certain types of leukaemia, if the leukaemia has spread to your brain or spinal cord, or if you've had an organ transplant. You also can't take part if you have serious or uncontrolled heart problems, especially those diagnosed recently, as these could increase risks during the study.

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.

Am I at least 12 years old?
Have I been diagnosed with Acute Myeloid Leukaemia (AML)?
Does my AML have specific gene changes (KMT2A, NPM1, NUP98, or NUP214)?
Is my general health, including liver and kidney function, considered good by my doctor?
Do I have any serious uncontrolled heart problems?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive Bleximenib in combination with other standard AML treatments. The type of standard treatment will depend on what your doctors think is best for you. Initially, the study will involve finding the right dose of Bleximenib, which means some participants might receive different amounts of the drug. After a safe dose is found, more people will join to further assess safety.

Throughout the study, you will have regular visits to the clinic for medical check-ups, blood tests, and other assessments to monitor your health and how the treatment is working. This will include keeping an eye on any side effects. You will need to take the study medication as directed by the clinical team. The total duration of your participation, including follow-up, will be explained in detail by the study team, but it will involve multiple appointments over a period of time.

Potential risks and benefits

Taking part in this study means you could potentially benefit from a new treatment for acute myeloid leukaemia that might be more effective, especially if other treatments haven't worked well for you. However, as with all new medicines, there are potential risks and side effects that are not yet fully known. The study team will carefully monitor your health for any side effects and manage them as best they can. You will be given detailed information about known risks. It's important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (32)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The University of Alabama at Birmingham
Verified postcode
Birmingham, United States
City of Hope
Verified postcode
Duarte, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
Albert Einstein College Of Medicine
Verified postcode
New York, United States
Novant Health
Verified postcode
Charlotte, United States
Novant Health Forsyth Medical Center
Verified postcode
Winston-Salem, United States
MD Anderson
Verified postcode
Houston, United States
Monash Medical Centre
Verified postcode
Clayton, Australia
Peter MacCallum Cancer Centre
Verified postcode
Melbourne, Australia
Westmead Hospital
Verified postcode
Westmead, Australia
Princess Margaret Cancer Centre University Health Network
Verified postcode
Toronto, Canada
Institut Paoli Calmettes
Verified postcode
Marseille, France

Common questions

What is AML?

AML stands for acute myeloid leukaemia, which is a fast-growing cancer that starts in the bone marrow, the soft tissue inside your bones where blood cells are made.

What does 'Phase 1' mean for a study?

Phase 1 means this is an early study where doctors are primarily checking if a new drug is safe for people and trying to find the best dose to use. It's not mainly about how well it treats the disease yet.

What is Bleximenib?

Bleximenib is a new experimental drug being tested in this study. Researchers hope it might help treat AML when combined with other medicines.

Will I know if I'm getting Bleximenib?

Yes, in this type of study, all participants will receive Bleximenib along with standard AML therapies. There isn't a placebo group receiving no active drug.

Can teenagers join this study?

Yes, teenagers aged 12 to 18 can join, but only if their AML has come back after treatment or hasn't responded to previous treatments.