RecruitingPHASE1INTERVENTIONAL

A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML

This research study is investigating a new oral medicine called CHM-029 for adults who have a specific type of acute myeloid leukaemia (AML). This AML either hasn't responded to previous treatments or has returned. The main goals are to carefully check how safe CHM-029 is, to find the most suitable dose, and to understand how the body uses and processes this new medicine. Researchers will also look at how effective it might be against the leukaemia. Participants will have regular check-ups, including blood tests and heart scans, to monitor their health and the medicine's effects. The study aims to gather important information that could help future patients.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

Charm Therapeutics

Enrolment target

Start

28 Sep 2026

Estimated completion

01 Mar 2029

AML (Acute Myeloid Leukemia)

What is this study about?

This study is looking into a new experimental medicine called CHM-029, which you take by mouth. It's for adults who have a type of blood cancer called Acute Myeloid Leukaemia (AML). Specifically, it's for those whose AML has certain genetic changes (called NPM1, KMT2A, or NUP98) and has either come back after treatment or didn't respond to standard treatments.

The main purpose of this early-stage study is to understand more about CHM-029. Researchers want to find out what dose is best, what possible side effects might occur, and how the medicine moves through and is used by the body. They will start with small doses and gradually increase them, carefully watching participants for any changes. This helps them learn about the medicine's safety and how well it's tolerated.

Taking part would involve regular visits to the hospital. During these visits, the study team will carry out various checks, including blood tests, heart scans (ECGs), and sometimes bone marrow tests. These are important for monitoring your health and seeing how you are responding to the treatment. This research is important because it could lead to new ways to treat AML in the future, especially for those who haven't responded well to current therapies.

Key takeaways

This study is testing a new oral medicine (CHM-029) for a specific type of AML.
It's for adults whose AML has certain genetic changes and hasn't responded to previous treatment or has come back.
The main goals are to find a safe dose and understand how the medicine works in the body.
Regular hospital visits, blood tests, and heart scans will be part of the study.
You can stop participating in the study at any time.
This is an early-stage study (Phase 1), focusing on safety.

Who may be eligible?

To join this study, you need to be at least 18 years old and have a specific type of Acute Myeloid Leukaemia (AML). This means your AML has either come back after treatment or hasn't responded to standard treatments, and it must have certain genetic changes (NPM1, KMT2A, or NUP98).

There are also some reasons why you might not be able to join. For example, if you have very high white blood cell counts that can't be controlled, or if your AML is only outside the bone marrow. You also couldn't join if you have ongoing issues from a past stem cell transplant, other serious medical conditions like certain heart problems, severe liver or kidney problems, or active infections like Hepatitis or HIV.

It's also not suitable if you have another type of leukaemia called acute promyelocytic leukaemia, or if you have certain heart rhythm issues. The study team will review all your medical information carefully to see if this study is the right 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?
Do you have AML that has come back or not responded to previous treatments?
Does your AML have the NPM1, KMT2A, or NUP98 genetic changes?
Do you have any serious heart conditions or severe liver/kidney problems?
Do you have uncontrolled high white blood cell counts?
Do you have other active cancers or infections (like Hepatitis or HIV) that need treatment?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would visit the study clinic regularly. These visits will involve various checks to monitor your health and how you respond to the medicine. You will have safety assessments, blood tests, and heart scans (ECGs). Sometimes, bone marrow evaluations will also be done. These tests help the doctors understand how the medicine is affecting you and your AML.

The medicine, CHM-029, will be given orally, meaning you'll take it by mouth. The study involves increasing dose levels, so the amount you take might change as the study progresses. The total duration of your participation will depend on how you respond to the treatment and the study plan, but you will be closely monitored throughout.

Before you decide, the study team will explain everything in detail, including the full schedule of visits and tests. You'll have plenty of opportunities to ask questions.

Potential risks and benefits

Participating in a study like this could offer potential benefits, such as access to a new experimental medicine for your AML that isn't yet widely available. This medicine might help to control your leukaemia. However, as with any new medicine, there are potential risks, including side effects that are not yet fully known. The study team will carefully monitor you for any side effects and will explain all known risks. Your safety is their priority. You are always free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

City of Hope Center Medical Center
Verified postcode
Duarte, United States· Recruiting
Yale - New Haven Hospital / Cancer Center
Verified postcode
New Haven, United States· Recruiting
START Midwest
Verified postcode
Grand Rapids, United States· Recruiting
Oxford University Hospitals NHS Foundation Trust
Verified postcode
Oxford, United Kingdom· 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, which is the spongy tissue inside your bones where blood cells are made.

What does 'investigational' medicine mean?

An 'investigational' medicine is a new drug that is still being studied to see if it's safe and effective. It hasn't been approved for general use yet.

Why are some people not allowed to join?

Certain health conditions or characteristics might make it unsafe for some people to take part, or they might make it difficult to properly understand how the new medicine works. This helps protect participants and ensures the study results are clear.

What is a Phase 1 study?

A Phase 1 study is usually the first time a new medicine is tested in humans. The main aim is to check its safety, find the best dose, and understand how the body handles it.

What are genetic changes like NPM1, KMT2A, or NUP98?

These are specific changes in the genetic material (DNA) within the leukaemia cells. Doctors look for these changes because they can tell us more about the AML and how it might respond to treatments.

How to find out more

Charm Study Contact

clinicaltrials@charmtx.com 312-682-0586 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.