AuthorisedPhase I and Phase II (Integrated)- First administration to humansInterventional

A Phase 1/2, Multicenter, First-in-Human, Dose Escalation and Expansion Study Evaluating CHM-029, as Monotherapy and in Combination with Standard Therapies in Participants with Relapsed or Refractory, or Newly Diagnosed Acute Myeloid Leukemia (AML) with NPM1 Mutations, KMT2A or NUP98 Rearrangements

Acute myeloid leukaemia (AML) is a fast-growing cancer of the white blood cells. While current treatments can help, leukaemia can sometimes return or resist standard therapy. This clinical trial is testing an experimental medicine called CHM-029 for the first time in humans. The trial is open to adults who have newly diagnosed AML, as well as those whose leukaemia has returned or stopped responding to past treatments. To join, a person's leukaemia cells must have specific genetic changes (such as NPM1, KMT2A, or NUP98). Because this is an early-stage study, the main goal is to test safety, discover potential side effects, and find the most suitable dose. Participants will receive CHM-029 on its own or combined with standard leukaemia therapies. Doctors will closely monitor their health, blood counts, and bone marrow to see if the treatment helps keep the leukaemia under control.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Charm Therapeutics Limited

Enrolment target

Start

30 Sep 2026

Acute Myeloid Leukemia

What is this study about?

Acute myeloid leukaemia (AML) is a serious cancer that affects blood-forming cells in the bone marrow. Although standard treatments like chemotherapy can be very effective, the cancer can sometimes come back after treatment or prove difficult to clear. In recent years, scientists have discovered that certain genetic changes inside leukaemia cells can influence how the disease behaves. This trial focuses on people whose leukaemia has specific changes, known as NPM1 mutations, or KMT2A and NUP98 rearrangements.

CHM-029 is a new experimental drug designed to target the way these altered leukaemia cells grow. Because this is a 'first-in-human' study, the medicine has not been given to people before. The first part of the trial involves a small number of patients testing very low doses. If safety checks look good, doctors gradually raise the dose for new groups to work out the safest and most effective amount.

Once a safe dose is established, researchers will study larger groups of patients. Some people will take CHM-029 on its own, while others will take it alongside standard AML medications, such as chemotherapy or targeted medicines like venetoclax. The goal is to see if combining these treatments can provide a stronger attack against cancer cells than standard treatment alone.

Studies like this are vital for creating new, gentler, and more precise cancer treatments. If CHM-029 is shown to be safe and helpful, it could become a valuable new option for people living with specific forms of AML in the future.

Key takeaways

Tests a new targeted drug (CHM-029) for acute myeloid leukaemia for the first time in people.
Open to adults with newly diagnosed, relapsed, or hard-to-treat AML.
Requires specific leukaemia gene changes: NPM1, KMT2A, or NUP98.
Aims primarily to find the safest dose and monitor any side effects.
Participation is voluntary, closely monitored, and can be stopped at any time.

Who may be eligible?

This study is open to adults aged 18 and over who have been diagnosed with acute myeloid leukaemia (AML). It includes people who are newly diagnosed, as well as those whose cancer has returned (relapsed) or did not respond to earlier treatments (refractory).

To be eligible, tests on your bone marrow or blood must show specific genetic markers in your leukaemia cells. These markers include an NPM1 mutation, or rearrangements involving the KMT2A or NUP98 genes. Your hospital medical team can check your routine test results to see if your leukaemia has these features.

You will also need to be well enough to carry out daily activities and attend frequent hospital appointments. People with severe heart, liver, or kidney problems, or active severe infections, may not be able to join. Anyone who is pregnant or breastfeeding cannot take part for safety reasons.

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.

I am aged 18 or older.
I have been diagnosed with acute myeloid leukaemia (AML).
My leukaemia is newly diagnosed, or has returned / not responded to past treatments.
My leukaemia cells have been confirmed to have an NPM1 mutation, or a KMT2A or NUP98 rearrangement.
I am well enough to attend regular hospital appointments and tests.
I am not pregnant or breastfeeding.

Answer every question to see your result.

What does participation involve?

Taking part involves receiving CHM-029 either as a tablet or an infusion, depending on the study group. Some participants will also take standard leukaemia treatments alongside it. In the early weeks, you will need to visit the hospital frequently for close health monitoring. Visits will involve regular blood tests, physical examinations, heart traces (ECGs), and occasional bone marrow biopsies to see how the leukaemia is responding. Treatment may continue for several months, provided the medicine is helping and side effects remain manageable. After treatment ends, the study team will continue to check on your recovery and health for a set follow-up period.

Potential risks and benefits

Because CHM-029 is being tested in humans for the first time, there is a risk of unexpected side effects, as well as known side effects linked to standard leukaemia medicines (such as tiredness, infection risk, or feeling sick). There is no guarantee that the new drug will improve your health. However, taking part may help control your leukaemia and will provide valuable knowledge to help future patients. Your safety is monitored very closely throughout, and you are entirely free to withdraw from the trial at any time without your regular NHS care being affected.

Locations (5)

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

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Unverified
France
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Unverified
Germany
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Unverified
Spain
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Unverified
Poland
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Unverified
Netherlands

Common questions

What does 'first-in-human' mean?

It means this experimental drug has been tested thoroughly in laboratories, but this study is the very first time it is being given to people.

Do I have to pay to take part in this trial?

No. In the UK, clinical trial medicines and study-related tests are provided free of charge, and travel expenses are often covered.

Can I leave the study if I change my mind?

Yes. Taking part is completely voluntary. You can stop participating at any point, and your doctors will continue to give you the best available standard care.

How do I know if my leukaemia has the right genetic markers?

Your hospital haematology team performs genetic tests on your blood and bone marrow as part of your normal care. They can tell you if your cells have NPM1, KMT2A, or NUP98 changes.

Will I receive a dummy drug (placebo)?

No. In an early-phase cancer trial like this, all participants receive active treatment. No one is given a placebo.