VA as Maintenance Therapy Post Allo-HSCT in MDS and AML

Venetoclax Combined With Azacytidine as Maintenance Therapy Post Allogeneic Hematopoietic Stem Cell Transplantation in Myelodysplastic Syndromes and Acute Myeloid Leukemia

The goal of this phase 2 trial is to test the safety and efficacy of Venetoclax combined with Azacytidine as Maintenance Therapy post Hematopoietic Stem Cell Transplantation in Myelodysplastic Syndromes and Acute Myeloid Leukemia.

Study Overview

• Status: Not yet recruiting
• Conditions: Acute Myeloid Leukemia
• Intervention / Treatment: Drug: Venetoclax combined with Azacytidine

Detailed Description

The investigators will evaluate the efficacy and safety of venetoclax combined with azacytidine as maintenance therapy post hematopoietic stem cell transplantation in myelodysplastic syndromes and acute myeloid leukemia. overall survival (OS), cumulative 1-year relapse- and progression-free survival (RPFS), time to relapse, relapse rate, incidence of acute and chronic GVHD, time to treatment discontinuation will be counted.

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Liren Qian, PhD; Phone Number: +861066947194; Email: qlr2007@126.com
• Study Contact Backup: Name: Yu Liu, PhD; Phone Number: +861066947192

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• The patient should, in the investigator's opinion, be able to meet all clinical trial requirements.
• The patient is willing and able to adhere to the study visit schedule and other protocol requirements.
• Patients aged ≥18 years with a diagnosis of MDS or AML according to World Health Organization criteria (WHO) who had undergone alloSCT with myeloablative or reduced-intensity conditioning regimens were eligible. Related and unrelated donors were permitted.
• Patients should be in morphologic complete remission (CR; ie, ≤5% bone marrow blasts) with absolute neutrophil counts ≥1.0×10^9 /L and platelets ≥ 75×10^ 9 /L before treatment.

Exclusion Criteria:

• Active or uncontrolled infections requiring systemic treatment within 14 days before enrollment.
• Any instability of systemic disease, including but not limited to severe cardiac, liver, kidney, or metabolic disease need therapy.
• Pregnant or lactating women.

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 Azacytidine; Patients were treated by venetoclax and azacytidine	Drug: Venetoclax combined with Azacytidine; Patients were treated by venetoclax and azacytidine: Venetoclax 100mg po qd d1, 200mg po qd d2, 400mg po qd d3-7; Azacytidine 75 mg/m2 subcutaneous injection qd d1-5.; Other Names: VA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Relapse Rate	Relapse following CR is defined as reappearance of leukemic blasts in the peripheral blood or the finding of more than 5% blasts in the BM, not attributable to another cause (eg, BM regeneration after consolidation therapy) or extramedullary relapse.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival (OS)	OS was defined as time from diagnosis to death from any cause or the last follow-up	through study completion, an average of 2 years
Progression Free Survival (PFS)	For patients in morphologic remission, documented relapse was considered progression. Relapse following complete remission(CR) is defined as reappearance of leukemic blasts in the peripheral blood or the finding of more than 5% blasts in the BM, not attributable to another cause (eg, BM regeneration after consolidation therapy) or extramedullary relapse.	1 year

Collaborators and Investigators

• Sponsor: Navy General Hospital, Beijing
• Investigators: Principal Investigator: Liren Qian, Navy General Hospital, Beijing

Study record dates

Study Major Dates

• Study Start (Estimated): September 1, 2024
• Primary Completion (Estimated): September 1, 2030
• Study Completion (Estimated): September 1, 2030

Study Registration Dates

• First Submitted: September 13, 2024
• First Submitted That Met QC Criteria: September 13, 2024
• First Posted (Actual): September 19, 2024

Study Record Updates

• Last Update Posted (Actual): October 1, 2024
• Last Update Submitted That Met QC Criteria: September 28, 2024
• Last Verified: September 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antimetabolites, Antineoplastic; Antimetabolites; Antineoplastic Agents; Venetoclax; Azacitidine
• Other Study ID Numbers: VAMHCT01

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