Recruiting NCT07238686
Venetoclax-containing Therapy Combined With Microtransplant for Intermediate-risk and Higher MDS
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Venetoclax, Azacitidine (AZA) or Decitabine (DAC), GPBMC infusion.
- Who it may be relevant to
- Registry conditions: Myelodysplastic Syndromes. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 2 Study of Venetoclax-containing Therapy in Combination With HLA-mismatched Mobilized Peripheral Blood Mononuclear Cell Infusion for Intermediate-risk and Higher Myelodysplastic Syndromes
Overview
This study aims to evaluate the safety and efficacy of a Venetoclax and hypomethylating agent-based regimen combined with infusion of HLA-mismatched donor G-CSF mobilized peripheral blood mononuclear cells (GPBMC) in patients with intermediate-risk and higher myelodysplastic syndromes who are ineligible for allogeneic hematopoietic stem cell transplantation.
Interventions
- Drug Venetoclax
Given PO. For the first cycle, dose of 100 mg on Day 1, 200 mg on Day 2, and 400 mg on Days 3-14. For other cycles, dose of 400 mg on Days 1-14. Note: The dose should be reduced to 100-200 mg daily if concomitant azole antifungals are required. - Drug Azacitidine (AZA) or Decitabine (DAC)
AZA: Given SC. Dose of 75 mg/m² on Days 1-7 of each cycle. DAC: Given IV. Dose of 20 mg/m² on Days 1-5 of each cycle. - Biological GPBMC infusion
HLA-mismatched donor GPBMCs are infused on Day 15.
Primary outcome measures
- Overall survival (OS) [Time frame: Measured up to 4 years after the last participant is enrolled]
- Overall response rate (ORR) [Time frame: Measured up to 2 years after the last participant is enrolled]
Secondary outcome measures (7)
- Modified overall response (mOR) [Time frame: Measured up to 2 years after the last participant is enrolled]
- Complete remission (CR) [Time frame: Measured up to 2 years after the last participant is enrolled]
- Partial remission (PR) [Time frame: Measured up to 2 years after the last participant is enrolled]
- CR with limited count recovery (CRL) [Time frame: Measured up to 2 years after the last participant is enrolled]
- CR with partial hematologic recovery (CRh) [Time frame: Measured up to 2 years after the last participant is enrolled]
- Progression-free survival (PFS) [Time frame: Measured up to 4 years after the last participant is enrolled]
- Treatment-related mortality (TRM) [Time frame: Measured up to 2 years after the last participant is enrolled]
Eligibility criteria
Inclusion criteria
- Age >=18 years, male or female, non-limited by race or ethnicity.
- Confirmed diagnosis of MDS according to the World Health Organization (WHO) 5th edition classification, based on histopathology and cytogenetics.
- Risk stratification according to the Revised International Prognostic Scoring System (IPSS-R) must place the patient in the intermediate-, high-, or very high-risk category.
- Not candidates for or refuse allogeneic hematopoietic stem cell transplantation.
- Adequate hepatic function including alanine transaminase (ALT) and aspartate aminotransferase (AST )<= 3 × upper limit of normal(ULN), and total bilirubin <= 1.5 × ULN.
- Adequate renal function including serum creatinine <= 2 × ULN or CrCl>= 40mL/min.
- LVEF measured by echocardiogram is within the normal range (LVEF > 50%).
- The subject must have one donor who is >= 18 years old and HLA matched at 0-7/10 loci (i.e., at least 3 HLA loci must be mismatched). In addition, the donor voluntarily donates hematopoietic stem cells and signs the consent form.
- Each subject (or his/her legal representatives) must sign the Informed Consent Form (ICF), indicating that he/she understands the purpose and procedures of research, and is willing to participate in research.
- Donor inclusion criteria: The donor meets the institution's criteria for related peripheral blood hematopoietic stem cell donors. The donor must be able to tolerate the cell separation and collection process, and sign the Informed Consent Form.
Exclusion criteria
- Uncontrolled infection or hemorrhage.
- Cardiovascular disease with clinical significance, such as uncontrolled or highly symptomatic cardiac arrhythmias, congestive heart failure, or myocardial infarction within 6 months prior to screening, or New York Heart Association (NYHA) function class 3 (moderate) or class 4 (severe) heart disease.
- Uncontrolled autoimmune disease or requiring immunosuppression treatment.
- History of severe blood infusion reaction.
- Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception.
- Psychiatric disorder or cognitive impairment that in the researcher's judgment would make the subject not likely to adhere to the protocol requirements.
- Major surgery within 4 weeks prior to enrollment.
- Life-threatening illness other than MDS or uncontrolled intercurrent illness.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital — Beijing
Identifiers
NCT: NCT07238686 · VEN-MST for MDS