Venetoclax Combined With Azactidine in the Treatment of ALAL

A Multicenter Prospective Clinical Study of Venetoclax Combined With Azactidine in the Treatment of Acute Leukaemias of Ambiguous Lineage

The prognosis of acute leukaemias of ambiguous lineage is poor. The effect of chemotherapy regimen and hematopoietic stem cell transplantation are still unclear. Therefore, we will explore new therapy to improve the remission rate of acute leukaemias of ambiguous lineage. Venetoclax can significantly improve the remission rate and prolong PFS and OS. At present, venetoclax combined with azacitidine or decitabine has become the preferred treatment regimen for elderly AML patients. It also shows a high response rate in relapsed/refractory AML or MDS patients. There are few clinical studies on the treatment of ALAL. The purpose of this study is to explore the efficacy and safety of venetoclax combined with azacitidine in the treatment of newly diagnosed ALAL patients.

Study Overview

• Status: Recruiting
• Conditions: Acute Leukemia of Ambiguous Lineage
• Intervention / Treatment: Drug: Venetoclax; Drug: azactidine

• Study Type: Interventional
• Enrollment (Estimated): 23
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Sheng-Li Xue, MD; Phone Number: 0086-0512-67781139; Email: slxue@suda.edu.cn

Study Locations

• China
  • Suzhou, China
    • Recruiting
    • The First Affliated Hospital of Soochow University
    • Contact: Sheng-Li Xue

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Patients aged ≥ 15 years.
2. Patients diagnosed with ALAL according to 5th edition of WHO Acute Leukaemias of Ambiguous Lineage diagnosis standard.
3. New diagnosed patients.
4. ECOG performance status score less than 3.
5. Expected survival time ≥3 months.
6. Patients without serious heart, lung, liver, or kidney disease.
7. Ability to understand and voluntarily provide informed consent.

Exclusion Criteria:

1. Patients who are allergic to the study drug or drugs with similar chemical structures.
2. Pregnant or lactating women, and women of childbearing age who do not want to practice effective methods of contraception.
3. Active infection.
4. Active bleeding.
5. Patients with new thrombosis, embolism, cerebral hemorrhage, or other diseases or a medical history within one year before enrollment.
6. Patients with mental disorders or other conditions whereby informed consent cannot be obtained and where the requirements of the study treatment and procedures cannot be met.
7. Liver function abnormalities (total bilirubin > 1.5 times the upper limit of the normal range, ALT/AST > 2.5 times the upper limit of the normal range or patients with liver involvement whose ALT/AST > 1.5 times the upper limit of the normal range), or renal anomalies (serum creatinine > 1.5 times the upper limit of the normal value).
8. Patients with a history of clinically significant QTc interval prolongation (male > 450 ms; female > 470 ms), ventricular heart tachycardia and atrial fibrillation, II-degree heart block, myocardial infarction attack within one year before enrollment, and congestive heart failure, and patients with coronary heart disease who have clinical symptoms and requiring drug treatment.
9. Surgery on the main organs within the past six weeks.
10. Drug abuse or long-term alcohol abuse that would affect the evaluation results.
11. Patients who have received organ transplants (excepting bone marrow transplantation).
12. Patients not suitable for the study according to the investigator's assessment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Venetoclax Combined With azactidine in the Treatment of Acute Leukaemias of Ambiguous Lineage; Venetoclax combined with azacitidine regimen. Venetoclax orally once daily (100 mg d1, 200 mg d2, 400 mg d3-28); azacitidine 75 mg/m2 subcutaneously once daily on days 1-7 .	Drug: Venetoclax; Venetoclax orally once daily (100 mg d1, 200 mg d2, 400 mg d3-28); Drug: azactidine; azacitidine 75 mg/m2 subcutaneously once daily on days 1-7

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate (ORR)	The overall response (complete remission, complete remission with incomplete blood count recovery) rate achieved after one or two courses (28 days) induction therapy by venetoclax combined azacitidine regimen.	At the end of Cycle 1 (each cycle is 28 days)
Complete Remission Rate (CRR)	The complete remission rate achieved after one or two courses (28 days) induction therapy by venetoclax combined azacitidine regimen.	At the end of Cycle 1 (each cycle is 28 days)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression-Free Survival (PFS)	It is measured from the date of entry into this trial to the date of progression or death.	1 year
Adverse events in hematological system	Record of adverse events in hematological system during and after designed venetoclax combined azacitidine regimen induction.	1 year
Overall survival (OS)	It is measured from the date of entry into this trial to the date of death from any cause; patients not known to have died at last follow-up are censored on the date they were last known to be alive.	1 year
Adverse events in other organs or systems	Record of adverse events in other organs or systems during and after designed venetoclax combined azacitidine regimen induction.	1 year

Collaborators and Investigators

• Sponsor: Sheng-Li Xue, MD
• Collaborators: The Second People's Hospital of Huai'an; Jining Medical University; Affiliated Hospital of Nantong University; Suzhou Hospital of Traditional Chinese Medicine; Pingdingshan first people's Hospital; Northern Jiangsu People's Hospital; The First Affiliated Hospital of Bengbu Medical University; Canglang Hospital of Suzhou; The Second Affiliated Hospital of Wannan Medical College

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2023
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): March 1, 2026

Study Registration Dates

• First Submitted: June 4, 2023
• First Submitted That Met QC Criteria: June 4, 2023
• First Posted (Actual): June 13, 2023

Study Record Updates

• Last Update Posted (Actual): December 16, 2025
• Last Update Submitted That Met QC Criteria: December 15, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia, Lymphoid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Biphenotypic, Acute; Antineoplastic Agents; venetoclax
• Other Study ID Numbers: SZALAL01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No