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

Charm Therapeutics

Status and phase

Enrolling

Phase 1

Conditions

AML (Acute Myeloid Leukemia)

Treatments

Drug: CHM-029

Study type

Interventional

Funder types

Industry

Identifiers

NCT07751991

2026-526409-14 (EudraCT Number)

CHM-029-01

Details and patient eligibility

About

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.

Full 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.

Enrollment

40 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

40 participants in 2 patient groups

CHM-029 Dose Escalation

Experimental group

Description:

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).

Treatment:

Drug: CHM-029

CHM-029 Backfill

Experimental group

Description:

Participants will receive CHM-029 orally at a dose level already evaluated by the Safety Review Committee (SRC).

Treatment:

Drug: CHM-029