A Study of Oral Venetoclax Tablets and Oral Azacitidine as Maintenance Therapy in Adult Participants With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy

Phase 3

Active, not recruiting

• Conditions: Acute Myeloid Leukemia (AML)
• Interventions: Drug: Venetoclax; Drug: Azacitidine

• Registration Number: NCT04102020
• Lead Sponsor: AbbVie

Brief Summary

This study will be conducted in two parts. Part 1 will be the Dose Confirmation portion to determine recommended Phase 3 dose (RPTD) of venetoclax in combination with azacitidine (AZA). Part 3 will be the Dose Finding portion to determine RPTD of venetoclax in combination with AZA. Part 2 and Part 3 Randomization of the study were removed.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-09-23
• Study First Submitted QC: 2019-09-23
• Study First Posted: 2019-09-25
• Study Start: 2020-03-26
• Primary Completion: 2022-09-29
• Disposition First Submitted: 2023-08-29
• Results First Submitted: 2025-07-29
• Status Verified: 2025-08
• Results First Submitted QC: 2025-08-19
• Last Update Submitted: 2025-08-19
• Results First Posted: 2025-08-20
• Disposition First Posted: 2025-08-20
• Last Update Posted: 2025-08-20
• Study Completion: 2026-06-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 112

Inclusion Criteria

• Diagnosis of newly diagnosed acute myeloid leukemia (AML).

• Participant meets the following disease activity criteria:

  • Confirmation of AML by World Health Organization (WHO) criteria (2016) and have confirmed complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following completion of intensive induction and consolidation chemotherapies.
  • Achieved first CR + CRi within 120 days of first dose of study drug or be no more than 75 days since last dose of intensive conventional (including both induction and consolidation) chemotherapies.
  • AML has intermediate or poor risk cytogenetics per National Comprehensive Cancer Network (NCCN) 2016 criteria.

• Eastern Cooperative Oncology Group (ECOG) performance status <= 2.

• Participant must have adequate hematologic, renal, and liver function laboratory values as described in the protocol.

Exclusion Criteria

• History of acute promyelocytic leukemia (APL).
• History of active central nervous system involvement with acute myeloid leukemia (AML).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Part 1: Dose Confirmation	Venetoclax	Participants will receive venetoclax once daily (QD) (Days 1-28) for up to 24 cycles, azacitidine (AZA) QD on Days 1-5 of each 28 day cycle for up to 6 cycles.
Part 1: Dose Confirmation	Azacitidine	Participants will receive venetoclax once daily (QD) (Days 1-28) for up to 24 cycles, azacitidine (AZA) QD on Days 1-5 of each 28 day cycle for up to 6 cycles.
Part 3 (Dose Finding): Dose Escalation	Venetoclax	Participants will receive venetoclax QD for up to 24 cycles, AZA QD on Days 1 to 14 of each 28-day cycle for up to 24 cycles to determine recommended phase 3 dose (RPTD).
Part 3 (Dose Finding): Dose Escalation	Azacitidine	Participants will receive venetoclax QD for up to 24 cycles, AZA QD on Days 1 to 14 of each 28-day cycle for up to 24 cycles to determine recommended phase 3 dose (RPTD).
Part 3 (Dose Finding): Safety Expansion	Venetoclax	Participants will receive venetoclax QD for up to 24 cycles, AZA QD on Days 1 to 14 of each 28-day cycle for up to 24 cycles at the RPTD.
Part 3 (Dose Finding): Safety Expansion	Azacitidine	Participants will receive venetoclax QD for up to 24 cycles, AZA QD on Days 1 to 14 of each 28-day cycle for up to 24 cycles at the RPTD.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Dose-Limiting Toxicities (DLTs) of Venetoclax in Combination With Azacitidine (AZA) (Part 1)	Up to 28 days (Cycle 1)	DLTs are any of the hematologic, nonhematologic toxicities, adverse events (AEs) occurring following administration of venetoclax as described in the protocol and evaluated by the Investigator and the sponsor.
Number of Participants With DLTs of Venetoclax in Combination With Oral AZA (Part 3 Dose Finding Portion)	Up to 28 days (Cycle 1)	DLTs are hematologic toxicities consisting of any Grade ≥ 3 neutropenia or thrombocytopenia lasting more than 7 days and nonhematologic toxicities as described in the protocol and evaluated by the Investigator and the Sponsor. In addition, AEs that lead to omitting \> 20% of the scheduled dose within the cycle is considered as a DLT unless clearly related to underlying disease. Treatment delay due to toxicity lasting greater than 14 days since the last dose of Venetoclax is also considered a DLT.

Trial Locations

Locations (156)

Mitchell Cancer Institute /ID# 216443; 🇺🇸 Mobile, Alabama, United States

Compassionate Cancer Care Research Group - Fountain Valley /ID# 216156; 🇺🇸 Fountain Valley, California, United States

University of California, Los Angeles /ID# 219149; 🇺🇸 Los Angeles, California, United States

Colorado Blood Cancer Institute /ID# 214280; 🇺🇸 Denver, Colorado, United States

St. Alphonsus Regional Medical /ID# 216424; 🇺🇸 Boise, Idaho, United States

Duplicate_Rush University Medical Center /ID# 218815; 🇺🇸 Chicago, Illinois, United States

University of Kentucky Markey Cancer Center /ID# 215048; 🇺🇸 Lexington, Kentucky, United States

Duplicate_Norton Cancer Institute /ID# 216401; 🇺🇸 Louisville, Kentucky, United States

Tulane Medical Center - New Orleans /ID# 218166; 🇺🇸 New Orleans, Louisiana, United States

Duplicate_Ochsner Clinic Foundation-New Orleans /ID# 214583; 🇺🇸 New Orleans, Louisiana, United States

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