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

NCT07047183 | Recruiting Phase 2

• 1 other identifier: 2025-HS-061
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 1

Brief Summary

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)

Conditions

Myelodysplastic Neoplasms; Transplantation

Outcome Measures

Primary Outcomes (1)

• 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

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

Secondary Outcomes (3)

• Overall Survival (OS)

  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)

  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）

  Through study completion, an average of 1 year

Study Arms (1)

venetoclax + Azacitidine

EXPERIMENTAL

Drug: VEN (Venetoclax)

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

Also known as: Azacitidine

venetoclax + Azacitidine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Newly diagnosed MDS confirmed by morphological and immunophenotypic analysis of bone marrow；
• Age ≥18 years, any gender；
• Bone marrow blasts ≥10%；
• IPSS-R score \>4.5；
• ECOG performance status 0-2；
• Scheduled for allogeneic hematopoietic stem cell transplantation (allo-HSCT)；
• Adequate major organ function：
• Cardiac: LVEF ≥50%
• Hepatic: Bilirubin ≤1.5×ULN
• AST/ALT ≤2.5×ULN
• Renal: Creatinine clearance ≥60 mL/min；
• Written informed consent provided by the patient or legally authorized representative.

You may not qualify if:

• Extramedullary disease involvement；
• Hypersensitivity to any study drugs；
• Severe cardiac disease, including congestive heart failure, myocardial infarction, and cardiac insufficiency;
• Concurrent malignant tumors of other organs, which can be enrolled if previously cured;
• Active tuberculosis or HIV infection;
• Concomitant hematologic disorders;
• Pregnancy or lactation;
• Inability to comply with protocol requirements;
• Concurrently participating in other clinical studies.

Sponsors & Collaborators

• Yehui Tan: lead

Study Sites (1)

The First Hospital of Jilin University

Changchun, Jilin, 130000, China

RECRUITING

MeSH Terms

Interventions

venetoclax; Azacitidine

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Central Study Contacts

Yehui Tan

CONTACT

8615948027438; yhtan@jlu.edu.cn

Yuying Li

CONTACT

8613944135650; lyying1001@jlu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 24, 2025
• First Posted: July 2, 2025
• Study Start: July 1, 2025
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): June 30, 2029
• Last Updated: August 8, 2025

Record last verified: 2025-08

Locations

CN (1)