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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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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | This cohort includes patients with intermediate-risk and higher MDS who are ineligible for or refuse allogeneic HSCT. Patients receive Venetoclax in combination with hypomethylating agent and HLA-mismatched donor GPBMC infusion. Treatment cycles are 28 days, repeated until disease progression or unacceptable toxicity. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival (OS) | OS is defined as the number of days from the date of study entry to the date of death. | Measured up to 4 years after the last participant is enrolled |
| Overall response rate (ORR) | 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. | Measured up to 2 years after the last participant is enrolled |
| Measure | Description | Time Frame |
|---|---|---|
| Modified overall response (mOR) | Modified OR is defined as CR+PR+CRL+CRh. | Measured up to 2 years after the last participant is enrolled |
| Complete remission (CR) | 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%. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Bo Cai, MD | Contact | +861066947168 | caibo2008@163.com | |
| Fei Peng, MD | Contact | +861066947180 | pengfei0118@foxmail.com |
| Name | Affiliation | Role |
|---|---|---|
| Bo Cai, MD | Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Department of Hematology, the Fifth Medical Center of Chinese PLA General Hospital | Recruiting | Beijing | 100071 | China |
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| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C579720 | venetoclax |
| D001374 | Azacitidine |
| D000077209 | Decitabine |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
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| 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. |
|
| Measured up to 2 years after the last participant is enrolled |
| Partial remission (PR) | 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. | Measured up to 2 years after the last participant is enrolled |
| CR with limited count recovery (CRL) | 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. | Measured up to 2 years after the last participant is enrolled |
| CR with partial hematologic recovery (CRh) | 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%. | Measured up to 2 years after the last participant is enrolled |
| Progression-free survival (PFS) | 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. | Measured up to 4 years after the last participant is enrolled |
| Treatment-related mortality (TRM) | TRM is defined as the death related to treatment instead of disease progression. | Measured up to 2 years after the last participant is enrolled |
| D011743 |
| Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |