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
- Study IRB#:2000043338
- Last Updated:09/16/2026
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.
Contact Us
For more information about this study, including how to volunteer, contact:
Farah Fasihuddin
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Trial Purpose and 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.
Eligibility Criteria
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
