NCT06191978

A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: M.D. Anderson Cancer Center · Academic / Other
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

To find a recommended dose of ASTX727 (cedazuridine/decitabine) in combination with venetoclax for pediatric patients with relapsed AML.

Detailed description

Primary Objectives 1. To determine the recommended dose of ASTX727 when given in combination with venetoclax in relapsed/refractory (R/R) pediatric participants with acute myeloid leukemia (AML). 2. To evaluate and describe the toxicities of ASTX727 and venetoclax administered in R/R AML pediatric population. 3. To characterize the pharmacokinetic (PK) and pharmacodynamic (PD) profile of ASTX727 alone and in combination with venetoclax in an R/R AML pediatric population. Secondary Objectives 1. To evaluate the potential for drug-drug interactions: effect of venetoclax on PK of ASTX727. 2. To define preliminary efficacy of ASTX727 when given in combination with venetoclax in pediatric participants. Efficacy will be based on Best Overall Response (BOR), including complete remission (CR), CR with incomplete blood count recovery, CR with incomplete hematologic recovery), and partial remission, overall survival (OS), event-free survival (EFS), and duration of response (DOR) of pediatric participants treated with this combination. Exploratory Objectives To quantify the number of pediatric participants transitioning to Hematopoietic stem cell transplant (HSCT), i.e., CR/CRi/CRh rate, HR. To identify biological markers of response to venetoclax and/or its combination with ASTX727. These effects could be at the molecular, cellular, or cytogenetic level.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	ASTX727	Given by PO
DRUG	Venetoclax	Given by PO

Timeline

• Start date: 2024-03-07
• Primary completion: 2028-12-31
• Completion: 2028-12-31
• First posted: 2024-01-05
• Last updated: 2026-02-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study