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

NCT07751991 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: CHM-029-012026-526409-14
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

AML (Acute Myeloid Leukemia)

Keywords

CHM-029; AML; Menin; leukemia; NPM1; KMT2A; NUP98; Charm

Outcome Measures

Primary Outcomes (6)

• 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.

  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.

  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

  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.

  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

  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

  Baseline through study completion, an average of 3 years

Secondary Outcomes (10)

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

  Baseline through study completion, an average of 3 years

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

  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)

  Baseline through study completion, an average of 3 years

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

  Baseline to study completion, an average of 3 years

• Composite complete remission rate (CRc)

  Baseline through study completion, an average of 3 years

Study Arms (2)

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).

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).

Drug: CHM-029

Interventions

CHM-029 DRUG

CHM-029 is administered orally.

CHM-029 Backfill; CHM-029 Dose Escalation

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Charm Therapeutics: lead

Study Sites (1)

START Midwest

Grand Rapids, Michigan, 49546, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Leukemia

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Central Study Contacts

Charm Study Contact

CONTACT

312-682-0586; clinicaltrials@charmtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2026
• First Posted: August 7, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): March 1, 2029
• Last Updated: August 7, 2026

Record last verified: 2026-08

Locations

US (1)