AML (Acute Myeloid Leukemia)Phase 1NCT07751991
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
A Phase 1 study testing CHM-029 in people with AML (Acute Myeloid Leukemia), run by Charm Therapeutics at 4 sites in 2 countries.
Trial summary
The goal of this study is to evaluate the safety of CHM-029, an investigational oral medicine, and to evaluate its activity in treating certain types of acute myeloid leukemia (AML) in adults. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML (Acute Myeloid Leukemia)
- What is being tested?
- CHM-029
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 28 Sep 2026to Mar 2029
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years old and above
- Relapsed or refractory (R/R) acute myeloid leukemia (AML) and has had treatment with any available standard therapies
- Positive for NPM1 mutation, or KMT2A or NUP98 rearrangements
Who cannot join
- White blood cell (WBC) count higher than 25,000 u/L that cannot be maintained below threshold with hydroxyurea treatment
- Extramedullary only AML
- Has current complications related to hematopoietic stem cell transplant (HSCT)
- Other cancers that require treatment
- Active Hepatitis or HIV infection
- Moderate hepatic or renal impairment
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- Acute promyelocytic leukemia
- Baseline prolongation of QT/QTc interval (≥ 470 ms) or additional risk factors for Torsades de Pointes (TdP)
- Congestive heart failure NYHA Class 3 or 4
- Central nervous system involvement refractory to intrathecal chemotherapy and/or standard cranial-spinal radiation
Trial locations and contacts
4 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 2 countries
United States3 sites, 3 recruiting
- Recruiting
City of Hope Center Medical Center
Duarte, California, 91010
Anthony S. Stein, MD
- Recruiting
START Midwest
Grand Rapids, Michigan, 49546
Andrew Sochacki, MD
- Recruiting
Yale - New Haven Hospital / Cancer Center
New Haven, Connecticut, 06510
Maximilian Stahl, MD
United Kingdom1 site, 1 recruiting
- Recruiting
Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 7LE
Paresh Vyas, Professor
About this study
Official title: A Phase 1/2, Multicenter, First-in-Human, Dose Escalation and Expansion Study Evaluating CHM-029 as Monotherapy in Participants With Relapsed or Refractory Acute Myeloid Leukemia (AML) With NPM1 Mutations, KMT2A or NUP98 Rearrangements
The goal of this study is to evaluate the safety of CHM-029, an investigational oral medicine, and to evaluate its activity in treating certain types of acute myeloid leukemia (AML) in adults. The main questions the study aims to answer are:
- What is an appropriate dose of CHM-029?
- What side effects may occur with CHM-029?
- How does the body process CHM-029?
Researchers will evaluate increasing dose levels of CHM-029 to better understand its safety and how the body responds to treatment.
Participants will visit the study clinic regularly for safety assessments, blood tests, electrocardiograms (ECGs), and bone marrow evaluations to monitor their health and response to treatment.
Read the detailed description
CHM-029-01 is a Phase 1/2, first-in-human, open-label, multicenter, dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antileukemic activity of orally administered CHM-029 in adults with relapsed or refractory acute myeloid leukemia (AML) with an NPM1 mutation, KMT2A rearrangement, or NUP98 rearrangement.
- Conditions
- AML (Acute Myeloid Leukemia)
- Being tested
- CHM-029
- Keywords
- CHM-029, AML, Menin, leukemia, NPM1, KMT2A, NUP98, Charm
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CHM-029Drug
oral
| Study group | Role | What they receive |
|---|---|---|
| CHM-029 Dose Escalation | Experimental | Participants will receive CHM-029 orally. Dose levels will be escalated based on dose limiting toxicities (DLTs) as evaluated by the Safety Review Committee (SRC). Receives: Drug: CHM-029 |
| CHM-029 Backfill | Experimental | Participants will receive CHM-029 orally at a dose level already evaluated by the Safety Review Committee (SRC). Receives: Drug: CHM-029 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with dose limiting toxicities (DLTs)
A DLT is defined as any Adverse Event (AE) which meets DLT criteria, not clearly due to the underlying disease or extraneous causes, that occurs within the DLT observation period.
Measured: Baseline through Day 28
Number of participants with adverse events (AEs)
An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Measured: Baseline through study completion, an average of 3 years
Number of participants with adverse events (AEs) by severity
Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 6.0
Measured: Baseline through study completion, an average of 3 years
Number of participants with laboratory value abnormalities and/or adverse events (AEs)
Number of participants with potentially clinically significant laboratory values.
Measured: Baseline through study completion, an average of 3 years
Rates of dose modification due to adverse events (AEs) according to NCI CTCAE
Safety and tolerability will be evaluated by dose interruption, modification, and discontinuation due to adverse events (AEs) according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v.6.0
Measured: Baseline through study completion, an average of 3 years
Maximum tolerated dose (MTD) and/or optimal biological dose (OBD) of CHM-029
Maximum tolerated dose or optimal biological dose will be determined by the sponsor based on the Safety Review Committee's recommendation considering the totality of the available clinical safety, clinical efficacy, pharmacokinetics (PK), and pharmacodynamic data
Measured: Baseline through study completion, an average of 3 years
Secondary outcomes (10)
Pharmacokinetics (PK) profile of CHM-029: Plasma concentrations
Maximum plasma concentrations of CHM-029 will be reported.
Measured: Baseline through study completion, an average of 3 years
Pharmacokinetic (PK) profile of CHM-029: Area under the curve
Pharmacokinetics will be calculated including the area under the plasma concentration versus time curve (AUC)
Measured: Baseline through study completion, an average of 3 years
Pharmacokinetic (PK) profile of CHM-029: Time of maximum concentration (Tmax) and half-life (T1/2)
Pharmacokinetic parameters will be calculated using standard non-compartmental techniques
Measured: Baseline through study completion, an average of 3 years
Complete remission and complete remission with partial hematological recovery (CR/CRh) rate
The CR/CRh rate is defined as the number of participants who achieved either CR or CRh at any of the post baseline visits divided by the number of participants in the analysis population.
Measured: Baseline to study completion, an average of 3 years
Composite complete remission rate (CRc)
The number of participants who achieve an overall response of complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), or complete remission with incomplete platelet recovery (CRp) at any post-baseline assessment, according to protocol-defined response criteria, divided by the number of participants in the analysis population.
Measured: Baseline through study completion, an average of 3 years
Duration of response (DOR)
DOR will be calculated among responders from the date of initial documentation of a response to the date of first documented evidence of relapse, as defined in the disease-specific response criteria, or death due to any cause, whichever occurs first.
Measured: Baseline through study completion, an average of 3 years
Morphological leukemia free state (MLFS) rate
The MLFS rate is defined as the number of participants who achieved MLFS at any post-baseline assessment, according to protocol-defined response criteria, divided by the number of participants in the analysis population.
Measured: Baseline through study completion, an average of 3 years
Best response rate
The number of participants who achieved a best overall response of CRc or MLFS, according to protocol-defined response criteria, divided by the number of participants in the analysis population.
Measured: Baseline through study completion, an average of 3 years
Overall survival (OS)
OS is defined from the date of first dose of study treatment to the date of death due to any cause.
Measured: Baseline through study completion, an average of 3 years
Event free survival (EFS)
EFS is defined as the number of days from the date of enrollment to the date of earliest evidence of relapse, treatment failure, or death.
Measured: Baseline through study completion, an average of 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2026
- Study start28 Sep 2026
- Primary completionDec 2027 (estimated)
- Study completionMar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myeloid Leukemia)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Charm Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07751991
- Sponsor's protocol ID
- CHM-029-01
- Other IDs
- 2026-526409-14
- Status last verified
- Oct 2026
- First submitted
- 30 Jul 2026
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided