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A Phase 1/2 study of CHM-029 alone and in combination drug regimen in adults with newly diagnosed, relapsed or refractory Acute Myeloid Leukemia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

Acute Myeloid Leukemia (AML) is a cancer that starts in the blood‑forming cells of the bone marrow and spreads through the bloodstream. Some people with AML have specific genetic changes called NPM1 mutations or rearrangements of the KMT2A or NUP98 genes, which can affect how the disease behaves and responds to treatment.

The study is testing a new oral capsule named CHM-029. This experimental drug will be given alone and together with medicines that are already used for AML, such as the infusion drug cytarabine, the oral drug venetoclax, the infusion drug azacitidine, the infusion drug daunorubicin, and the oral antifungal medication posaconazole. The goal is to learn how the new capsule works in the body and whether it can be combined safely with these standard treatments.

The purpose of the trial is to evaluate safety and to find the dose of CHM-029 that can be given without causing unacceptable side effects, often called the maximum tolerated dose or optimal biological dose. Participants will start with a low dose that is gradually increased while doctors closely watch for any adverse reactions through regular check‑ups, blood tests, and other routine assessments. If the drug is well tolerated, some participants may receive it together with the standard chemotherapy regimen, and the study will continue for several weeks to months to observe any signs that the leukemia is responding.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Start of treatment
On the first day after joining the study, you will receive the investigational medicine chm-029 in capsule form taken by mouth.
The capsule is part of a treatment plan that may also include standard medicines such as cytarabine (given by infusion), venetoclax (taken by mouth), azacitidine (given by infusion), daunorubicin (given by infusion), and posaconazole (taken by mouth).
The exact amount, how often each medicine is taken, and how long the treatment lasts are decided by the study doctors and will be explained to you before you start.
Step 2
Regular medication administration
You will follow a schedule for each medicine:
chm-029: taken orally as a capsule at the frequency set by the study team.
cytarabine and azacitidine: given through an intravenous line (infusion) on the days specified in the protocol.
venetoclax and posaconazole: taken by mouth according to the prescribed timing.
daunorubicin: also given by infusion on the days required by the treatment plan.
Step 3
Safety monitoring visits
Throughout the study you will attend regular clinic visits.
At each visit blood samples and other laboratory tests will be taken to check how your body is responding to the medicines.
The study staff will record any side effects and may adjust the medication if needed.
Step 4
Dose escalation
The study is designed to find the highest dose of chm-029 that can be given safely.
If you tolerate the initial dose without significant side effects, the dose may be increased in later cycles according to a predefined schedule.
Each increase will be followed by additional safety checks before the next dose is given.
Step 5
Repeat treatment cycles
The combination of medicines is given in repeated cycles as described in the study protocol.
Each cycle includes the same set of medicines and monitoring procedures.
The number of cycles you will receive depends on your response and the study guidelines.
Step 6
Completion of treatment
When the planned number of cycles is finished or if the study doctors decide to stop treatment, you will stop taking the study medicines.
A final set of assessments will be performed to evaluate the overall effect of the treatment.
Step 7
Post‑treatment follow‑up
After treatment ends, you will continue to have periodic visits for several months.
These visits will monitor your health status, disease progression, and overall survival.
The follow‑up period helps researchers understand the long‑term impact of the therapy.

Who can join the trial?

12 criteria

Written informed consent must be signed, showing you understand the study and agree to take part, and it must include privacy protections required by law.
You must be at least 18 years old when you sign the consent form.
You must have a diagnosis of Acute Myeloid Leukemia (AML) that has been confirmed by standard laboratory tests used by the World Health Organization (WHO) criteria (these are widely accepted rules for identifying this type of cancer).
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Your disease must fit into one of the following groups:
- Relapsed AML – the cancer returned after you had a complete remission (the disease disappeared).
- Refractory AML – the cancer did not go into remission after your most recent treatment.
- Newly diagnosed AML that cannot receive intensive chemotherapy because you are 75 years or older, or you have certain health problems such as serious heart failure, reduced heart pumping ability (ejection fraction ≤ 50 %), chronic stable chest pain, significant lung disease, reduced kidney function, or moderate liver problems.
- Newly diagnosed AML that is eligible for intensive chemotherapy.
- You must have one of the specific genetic changes tested for in the study: a NPM1 mutation, or a rearrangement of the KMT2A or NUP98 genes. Patients with a KMT2A partial tandem duplication or amplification are not allowed.
- Your overall health, measured by the Eastern Cooperative Oncology Group (ECOG) performance status, must be 2 or lower for most groups, or 3 or lower for the group that includes patients who cannot tolerate intensive chemotherapy. (ECOG is a simple scale that describes how well you can carry out daily activities, from fully active (0) to completely disabled (5).)
- If you have relapsed or refractory disease, you must wait at least 2 weeks after finishing any chemotherapy that kills cells (except hydroxyurea) before starting the study drug, or wait a period equal to five times the “half‑life” of any prior experimental or non‑cell‑killing medication.
- You must be able to take the study medication by mouth (oral administration), such as a tablet or liquid.
- You must agree to follow the study’s reproductive requirements, which usually means using effective birth control or other measures to prevent pregnancy during the trial.

Who cannot join the trial?

18 criteria

If your white blood cell count is higher than 25,000 per microliter and cannot be lowered with the medicine hydroxyurea, you cannot join the study. (White blood cell (WBC) count measures the number of immune cells in the blood; hydroxyurea is a drug that can reduce this count.)
If your leukemia is only found outside the bone marrow (called extramedullary AML), you are not eligible.
If you have had a stem cell transplant and you currently have serious graft‑versus‑host disease that needs systemic treatment, or you have persistent non‑blood‑related side effects of grade 2 or higher from the transplant, you cannot participate. (Graft‑versus‑host disease is when donated immune cells attack the recipient’s body; grade 2 indicates moderate severity.)
If you have another cancer that still needs treatment, you are excluded.
If you have an uncontrolled bacterial, viral, or fungal infection at the time you would start the study, you cannot join.
If you have an active infection with hepatitis B or hepatitis C viruses, you are not eligible.
If you test positive for HIV‑1 or HIV‑2, you cannot take part in the trial.
If your heart’s electrical recovery time (QT/QTc interval) is very long – a measured value of 470 milliseconds or more on three separate tests – you are excluded. (QT/QTc interval is a measure on an electrocardiogram that reflects how quickly the heart repolarizes.)
If you have other risk factors for a dangerous heart rhythm called torsades de pointes (TdP), such as heart failure, low potassium that does not improve with treatment, or a family history of Long QT syndrome, you cannot join.
If you are in Cohort 2, Stage 2 and have an IDH1 mutation but are not already eligible for an approved IDH1‑inhibitor drug used with azacitidine, you are excluded.
If leukemia has spread to the brain or spinal fluid and does not respond to intrathecal chemotherapy (medicine delivered into the spinal fluid) or standard cranial‑spinal radiation, you cannot participate.
If your liver enzymes (AST or ALT) are more than three times the normal upper limit, you are excluded. (AST and ALT are blood tests that show liver health.)
If you have acute promyelocytic leukemia, a specific subtype of AML, you are not eligible.
If the study doctor believes any condition you have would interfere with the study, put you at significant risk, or make the study results difficult to interpret, you will be excluded.
If you have moderate to severe heart failure (NYHA Class 3 or 4) now or in the past, unless a recent heart ultrasound (echocardiogram) shows a left‑ventricle pumping ability (ejection fraction) of at least 45%, you cannot join. (NYHA Class describes the severity of heart failure symptoms; ejection fraction measures how well the heart pumps blood.)
If your kidney function is moderately reduced, meaning an estimated glomerular filtration rate (eGFR) below 60 mL/min, you are excluded. (eGFR estimates how well the kidneys filter waste.)
If you have moderate liver problems, defined as total bilirubin more than 1.5 times the normal upper limit, you cannot participate, except if you have Gilbert’s syndrome with total bilirubin up to three times normal, normal direct bilirubin, and liver enzymes (AST, ALT) no more than three times normal, and no other liver disease. (Bilirubin is a substance processed by the liver; Gilbert’s syndrome is a mild, inherited condition that can raise bilirubin levels.)
If you are in Cohort 3 and have an FLT3‑ITD mutation with a variant allele frequency (VAF) of 5% or higher, you are excluded. (FLT3‑ITD mutation is a genetic change in leukemia cells; VAF indicates how many of the cells carry that mutation.)

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Centre Hospitalier Universitaire De Caen Normandie
Caen, France
Verified
Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
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Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands

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Investigated drugs

CYTARABINE
is a chemotherapy drug given through an IV drip. It works by stopping cancer cells from copying their DNA, which helps to kill the leukemia cells. In this trial it is used as part of the standard background treatment for acute myeloid leukemia (AML).
VENETOCLAX
is an oral medication that blocks a protein called BCL‑2, which many leukemia cells use to avoid death. By turning off this survival signal, the drug helps the cancer cells die. It is included as a standard background therapy in the study.
AZACITIDINE
is given by intravenous infusion. It helps abnormal blood‑forming cells mature and die, slowing the growth of leukemia. This drug is also part of the standard background treatment used in the trial.
CHM-029
is an experimental capsule taken by mouth. The study is testing this new drug alone and together with the standard AML medicines to see if it is safe and if it can improve outcomes for patients whose leukemia has specific genetic changes.
DAUNORUBICIN
is a chemotherapy agent delivered through an IV infusion. It works by damaging the DNA inside cancer cells, which leads to their death. It is used as a standard background therapy in the trial.
POSACONAZOLE
is an oral antifungal medicine that helps prevent serious fungal infections, which can be a risk when patients receive strong chemotherapy for AML. It is part of the standard background care in the study.

What is already known about the treatment

Cytarabine
Cytarabine is given by intravenous infusion and is an approved chemotherapy drug for acute myeloid leukemia (AML). It is a nucleoside analog that blocks the building of DNA, which stops cancer cells from multiplying. In the medical literature it is listed as a standard antimetabolite used in many AML treatment regimens. It belongs to the class of antimetabolite chemotherapy agents.
Venetoclax
Venetoclax is taken orally as a tablet and is a licensed therapy for certain blood cancers, including AML when used with other drugs. It works by attaching to the BCL‑2 protein inside cells, freeing the cell’s natural death program and allowing cancer cells to die. It is described in current medical sources as a targeted BCL‑2 inhibitor. Its pharmacological class is a selective BCL‑2 inhibitor.
Azacitidine
Azacitidine is administered by intravenous infusion and is an approved treatment for AML and related blood disorders. It adds small chemical groups to DNA and RNA, which can turn back on genes that help control cell growth. The drug is recognized in the literature as a hypomethylating agent used in standard care. It belongs to the class of DNA‑methyltransferase inhibitors.
Daunorubicin
Daunorubicin is given by intravenous infusion and is a well‑established chemotherapy for AML. It slips between DNA strands and creates free radicals that damage the DNA, leading to cancer cell death. It is listed in medical references as a core component of many AML regimens. Its classification is an anthracycline antibiotic chemotherapy.
Posaconazole
Posaconazole is taken orally, usually as a tablet, and is an approved antifungal used to prevent infections in people with weak immune systems, such as those undergoing AML treatment. It blocks a key fungal enzyme called lanosterol 14α‑demethylase, stopping the fungus from making its cell membrane. Current guidelines describe it as a broad‑spectrum azole antifungal. It is classified as an azole antifungal agent.
CHM-029
CHM-029 is an oral capsule being tested for the first time in people with AML that has specific genetic changes. It is not yet approved and is currently in early clinical trials to determine safety and the right dose. Early data suggest it may act on a unique molecular pathway that helps stop the growth of leukemia cells, but the exact mechanism is still under study. It is considered an experimental anticancer agent.

Investigated diseases

Acute Myeloid Leukemia - Acute Myeloid Leukemia is a blood cancer that begins in the bone marrow, where blood cells are produced. It causes the marrow to make many immature white blood cells that cannot function normally. These abnormal cells grow quickly and push out healthy blood cells, leading to low red cells, low platelets, and weakened immunity. The disease can spread from the marrow into the bloodstream and reach organs such as the spleen and liver. It typically progresses rapidly, requiring prompt medical attention.