NCT06113289

A Phase 1B/2A Trial of Combination of ASTX727 With ASTX029 in Acute Myeloid Leukemia

• Status: Withdrawn
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

To find the recommended dose of the study drugs ASTX727 and ASTX029 that can be given to patients with relapsed/refractory AML. The goal of Part 2 of the study is to learn if the dose of study drugs found in Part 1B can help to control AML.

Detailed description

Primary Objectives: To determine the safety and recommended phase 2 dose (RP2D) of ASTX727 in combination with ASTX029 in patients with relapsed/refractory AML. Secondary Objectives: * To assess the CR+CRi+PR and MLFS rate within 6 cycles of treatment initiation of ASTX727 in combination with ASTX029 in patients with relapsed refractory AML. * To determine the duration of response (DOR), event-free survival (EFS), overall survival (OS), minimal/measurable residual disease (MRD) status at response and best MRD response attained by flow-cytometry, 4- and 8-week mortality * To investigate correlations of response to these combinations with a pre- therapy, on-therapy, and progression 81-gene panel of gene mutations in AML. Exploratory Objectives: * To investigate possible relationships between response and non-response to the combinations with myeloid mutation panel. * To identify leukemic subpopulations and how their signaling state in disease relates to clinical outcomes by Flow cytometry or CyTOF (mass cytometry) on patients' bone marrow samples and/or peripheral blood baseline, on treatment, remission and relapse and potentially other time-points on study. * To store and/or analyze surplus blood or tissue including bone marrow, if available, for potential future exploratory research into factors that may influence development of AML and/or response to the combination (where response is defined broadly to include efficacy, tolerability or safety).

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	ASTX727	Given by PO
DRUG	ASTX029-01	Given by PO

Timeline

• Start date: 2024-02-08
• Primary completion: 2025-03-06
• Completion: 2025-03-06
• First posted: 2023-11-02
• Last updated: 2025-03-12

Regulatory

• FDA-regulated drug study