Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes

Phase 2RecruitingNCT07047183

Yehui Tan46 enrolled

Overview

A Single-Arm, Prospective Clinical Study of Venetoclax Combined with Azacitidine Followed by Bridging Transplantation in Patients with High-Risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2)

This is a single-center, single-arm, prospective study investigating pre-transplant bridging therapy in patients aged ≥18 years with higher-risk MDS-IB2. The study plans to enroll 46 eligible patients. Participants will receive Venetoclax combined with Azacitidine, administered in 28-day cycles for 1 to 2 cycles. Treatment response will be assessed according to the IWG 2023 HR-MDS response criteria.Patients achieving modified Composite Complete Remission (mCRc: CR or CR-equivalent + CRL + CRh) after Cycle 1 will proceed directly to transplantation. Patients not achieving mCRc will receive a second cycle of therapy. All patients will undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) within 3 months after completing Cycle 2, regardless of mCRc status. Patients unable to proceed to transplantation will receive standard institutional care and undergo follow-up.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myelodysplastic Neoplasms Transplantation

Interventions

VEN (Venetoclax)DRUG

Enrolled patients will receive: Venetoclax: 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. Azacitidine: 75 mg/m² on Days 1-7. Cycle duration: 28 days Total cycles: 1 to 2 cycles

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow； 2. Age ≥18 years, any gender； 3. Bone marrow blasts ≥10%； 4. IPSS-R score \>4.5； 5. ECOG performance status 0-2； 6. Scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT)； 7. Adequate major organ function： * Cardiac: LVEF ≥50% * Hepatic: Bilirubin ≤1.5×ULN * AST/ALT ≤2.5×ULN * Renal: Creatinine clearance ≥60 mL/min； 8. Written informed consent provided by the patient or legally authorized representative. Exclusion Criteria: 1. Extramedullary disease involvement； 2. Hypersensitivity to any study drugs； 3. Clinically significant hepatic/renal dysfunction exceeding inclusion thresholds； 4. Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency; 5. Concurrent malignant tumors of other organs, which can be enrolled if previously cured; 6. Active tuberculosis or HIV infection; 7. Concomitant hematologic disorders; 8. Pregnancy or lactation; 9. Inability to comply with protocol requirements; 10. Concurrently participating in other clinical studies.

Locations (1)

The First Hospital of Jilin University

Changchun, Jilin, China

RECRUITING

Outcomes

Primary Outcomes

2-Year Relapse-Free Survival(2-RFS)

Defined as the time from enrollment in this study to the occurrence of relapse. For patients without relapse, the time is calculated until death from any cause or the time of the last follow-up

Time frame: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.

Secondary Outcomes

Overall Survival (OS)

Defined as the time from enrollment in this study to death from any cause. If the exact date of death is unknown, the time of death is defined as the last contact date. If the patient is still alive, the last observed time will be used, and the patient's OS will be considered for analysis.

Time frame: From date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 48 months.

Cumulative Relapse Rate (CIR)

Defined as the proportion of patients who experience disease relapse from the time of enrollment in this study.

Time frame: rom date of index treatment to death, disease progression, or end of study, whichever came first, assessed up to 24 months.

Composite Complete Response Rate（mCRc）

Complete Remission (CR) or CR-equivalent+ CR with partial hematologic recovery (CRL) + CR with limited hematologic recovery (CRh)

Time frame: Through study completion, an average of 1 year

Central Contacts

Yehui Tan

CONTACT

8615948027438 yhtan@jlu.edu.cn

Yuying Li

CONTACT

8613944135650 lyying1001@jlu.edu.cn

Data from ClinicalTrials.gov

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