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NCT Number: NCT07238686

Venetoclax-containing Therapy Combined With Microtransplant for Intermediate-risk and Higher MDS

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.

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Key information

Conditions

Myelodysplastic Syndromes Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Venetoclax

Drug

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.

Azacitidine (AZA) or Decitabine (DAC)

Drug

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.

GPBMC infusion

Biological

HLA-mismatched donor GPBMCs are infused on Day 15.

Primary outcomes

  1. Overall survival (OS)

    Time frame: Measured up to 4 years after the last participant is enrolled

    OS is defined as the number of days from the date of study entry to the date of death.

  2. Overall response rate (ORR)

    Time frame: Measured up to 2 years after the last participant is enrolled

    ORR is defined as complete remission (CR) (or CR equivalent) + partial remission (PR) + CR with limited count recovery (CRL) + CR with partial hematologic recovery (CRh) + hematologic improvement (HI) according to IWG 2023 criteria.

Secondary outcomes

  1. Modified overall response (mOR)

    Time frame: Measured up to 2 years after the last participant is enrolled

    Modified OR is defined as CR+PR+CRL+CRh.

  2. Complete remission (CR)

    Time frame: Measured up to 2 years after the last participant is enrolled

    CR is defined as absolute neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, Hb ≥10 g/dL, bone marrow with < 5% blasts, and PB blasts 0%.

  3. Partial remission (PR)

    Time frame: Measured up to 2 years after the last participant is enrolled

    PR is defined as decrease of at least 50% in the percentage of blasts still ≥5% in the bone marrow, peripheral blood neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, Hb ≥10 g/dL.

  4. CR with limited count recovery (CRL)

    Time frame: Measured up to 2 years after the last participant is enrolled

    CRL is defined as bone marrow with less than 5% blasts, PB blasts 0%, and PB only meeting 1 or 2 of the following: absolute neutrophil count > 1 × 109/L, platelet count > 100 × 109/L, Hb ≥10 g/dL.

  5. CR with partial hematologic recovery (CRh)

    Time frame: Measured up to 2 years after the last participant is enrolled

    CRh is defined as bone marrow with less than 5% blasts, and PB not meeting criteria for CR or CRL, no Hb threshold required, platelets ≥50 × 109/L, neutrophils ≥0.5 × 109/L, blasts 0%.

  6. Progression-free survival (PFS)

    Time frame: Measured up to 4 years after the last participant is enrolled

    PFS is defined as the number of days from the date of study entry to the date of the first of the following events: progressed disease; relapse from CR (or CR equivalent), PR, CRL, CRh, or HI; death from any cause.

  7. Treatment-related mortality (TRM)

    Time frame: Measured up to 2 years after the last participant is enrolled

    TRM is defined as the death related to treatment instead of disease progression.

Study contacts

Contact information is provided by the study sponsor or research team.

Bo Cai, MD

CONTACT

[email protected]

+861066947168

Fei Peng, MD

CONTACT

[email protected]

+861066947180

Sponsors and collaborators

Lead sponsor

Beijing 302 Hospital

Other

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2030
Study completion
2031
First posted
Nov 20, 2025
Registry last updated
Nov 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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