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NCT Number: NCT07751991

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

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.

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Key information

Conditions

AML (Acute Myeloid Leukemia) Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Neoplasms Neoplasms by Histologic Type

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

START Midwest

Grand Rapids, Michigan, 49546, United States

Location status: Recruiting

Location contact

Andrew Sochacki, MD

CONTACT

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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
  • 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

Treatment and study plan

CHM-029

Drug

CHM-029 is administered orally.

Primary outcomes

  1. Number of participants with dose limiting toxicities (DLTs)

    Time frame: Baseline through Day 28

    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.

  2. Number of participants with adverse events (AEs)

    Time frame: Baseline through study completion, an average of 3 years

    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.

  3. Number of participants with adverse events (AEs) by severity

    Time frame: Baseline through study completion, an average of 3 years

    Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 6.0

  4. Number of participants with laboratory value abnormalities and/or adverse events (AEs)

    Time frame: Baseline through study completion, an average of 3 years

    Number of participants with potentially clinically significant laboratory values.

  5. Rates of dose modification due to adverse events (AEs) according to NCI CTCAE

    Time frame: Baseline through study completion, an average of 3 years

    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

  6. Maximum tolerated dose (MTD) and/or optimal biological dose (OBD) of CHM-029

    Time frame: Baseline through study completion, an average of 3 years

    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

Secondary outcomes

  1. Pharmacokinetics (PK) profile of CHM-029: Plasma concentrations

    Time frame: Baseline through study completion, an average of 3 years

    Maximum plasma concentrations of CHM-029 will be reported.

  2. Pharmacokinetic (PK) profile of CHM-029: Area under the curve

    Time frame: Baseline through study completion, an average of 3 years

    Pharmacokinetics will be calculated including the area under the plasma concentration versus time curve (AUC)

  3. Pharmacokinetic (PK) profile of CHM-029: Time of maximum concentration (Tmax) and half-life (T1/2)

    Time frame: Baseline through study completion, an average of 3 years

    Pharmacokinetic parameters will be calculated using standard non-compartmental techniques

  4. Complete remission and complete remission with partial hematological recovery (CR/CRh) rate

    Time frame: Baseline to study completion, an average of 3 years

    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.

  5. Composite complete remission rate (CRc)

    Time frame: Baseline through study completion, an average of 3 years

    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.

  6. Duration of response (DOR)

    Time frame: Baseline through study completion, an average of 3 years

    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.

  7. Morphological leukemia free state (MLFS) rate

    Time frame: Baseline through study completion, an average of 3 years

    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.

  8. Best response rate

    Time frame: Baseline through study completion, an average of 3 years

    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.

  9. Overall survival (OS)

    Time frame: Baseline through study completion, an average of 3 years

    OS is defined from the date of first dose of study treatment to the date of death due to any cause.

  10. Event free survival (EFS)

    Time frame: Baseline through study completion, an average of 3 years

    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.

Study contacts

Contact information is provided by the study sponsor or research team.

Charm Study Contact

CONTACT

[email protected]

312-682-0586

Sponsors and collaborators

Lead sponsor

Charm Therapeutics

Industry

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2027
Study completion
2029
First posted
Aug 7, 2026
Registry last updated
Aug 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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