Actively Recruiting
Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes
Led by Yehui Tan · Updated on 2025-08-08
46
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a treatment approach for adults aged 18 and older with higher-risk Myelodysplastic Neoplasms with Increased Blasts 2 (MDS-IB2). This single-arm, prospective study investigates the use of Venetoclax combined with Azacitidine as a bridging therapy before allogeneic hematopoietic stem cell transplantation (allo-HSCT). The goal is to assess treatment response and survival outcomes in this patient population. Participants will receive Venetoclax and Azacitidine in 28-day cycles, with dosing starting at 100 mg of Venetoclax on Day 1 and escalating to 400 mg by Days 3-14, alongside Azacitidine at 75 mg/m² on Days 1-7. They may receive one or two cycles depending on their treatment response. Those achieving a modified Composite Complete Remission after the first cycle proceed directly to transplantation, while others receive a second cycle before undergoing allo-HSCT within three months. Patients unable to proceed to transplantation will receive standard care and monitoring. During the study, participants will be closely monitored for treatment response using established criteria and followed for outcomes such as relapse-free survival over two years. Additional measures include overall survival, relapse rates, and complete response rates assessed up to four years. Safety and organ function will be evaluated, and follow-up care will continue after transplantation or standard treatment as appropriate.
CONDITIONS
Brief Title
Clinical Study of Venetoclax Combined With Azacitidine as Bridging Therapy Prior to Hematopoietic Stem Cell Transplantation in Patients With Higher-Risk Myelodysplastic Syndromes
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Newly diagnosed Myelodysplastic Syndrome confirmed by bone marrow analysis
- Age 18 years or older, any gender
- Bone marrow blasts of 10% or higher
- IPSS-R score greater than 4.5
- ECOG performance status between 0 and 2
- Scheduled for allogeneic hematopoietic stem cell transplantation
- Adequate organ function: Left ventricular ejection fraction at least 50%, bilirubin no more than 1.5 times upper limit, AST/ALT no more than 2.5 times upper limit, creatinine clearance at least 60 mL/min
- Provided written informed consent
You will not qualify if you...
- Presence of extramedullary disease involvement
- Hypersensitivity to study drugs
- Significant liver or kidney dysfunction exceeding inclusion limits
- Severe heart disease such as congestive heart failure or myocardial infarction
- Active other cancers unless previously cured
- Active tuberculosis or HIV infection
- Other blood disorders
- Pregnancy or breastfeeding
- Inability to follow study procedures
- Participation in other clinical trials concurrently
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 to 2 cycles of 28 days each
Participants receive Venetoclax combined with Azacitidine as bridging therapy prior to hematopoietic stem cell transplantation. Treatment consists of 1 to 2 cycles of 28 days each. Participants achieving remission after the first cycle proceed directly to transplantation, while others receive a second cycle.
Visits during each 28-day cycle as per treatment administration schedule
Duration - Up to 3 months post-treatment for transplantation
All participants undergo allogeneic hematopoietic stem cell transplantation within 3 months after completing up to 2 cycles of therapy. Participants unable to proceed to transplantation receive standard institutional care and follow-up.
Visits as required for transplantation and immediate care
Duration - Up to 48 months
Participants are monitored following transplantation or standard care to assess outcomes including relapse-free survival and overall survival.
Periodic visits for up to 48 months
Trial Site Locations
Total: 1 location
1
The First Hospital of Jilin University
Changchun, Jilin, China, 130000
Actively Recruiting
Research Team
Y
Yehui Tan
Y
Yuying Li
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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