Safety and Efficacy of Unrelated Umbilical Cord Blood Microtransplantation in Patients With Higher-risk MDS

NCT06109064 | Unknown Phase 2

• 1 other identifier: UCB-MST&MDS
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

This study aimed to evaluate the safety and efficacy of unrelated umbilical cord blood microtransplantation in the treatment of above-mentioned MDS patients by observing the factors related to the efficacy and adverse reactions.

Conditions

Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (2)

• Complete response (CR) rate

  CR rate is defined as the percentage of patients who met the following conditions: Myelogram: naïve cells less than 5% (at least 200 nucleated cells); Hemogram: absolute neutrophil count \> 1.0×10\^9/L, platelet count \> 100×10\^9/L; Clinical diagnosis: no symptoms and signs caused by leukemia infiltration, and patients do not rely on blood transfusion.

  28±7 days

• Hematopoietic recovery time

  The time of absolute neutrophil count\>0.5×10\^9/L and platelet count \>30×10\^9/L for 3 consecutive days.

  28±7 days

Secondary Outcomes (4)

• Time to Progression（TTP）

  1 year

• Disease Free Survival（DFS）

  1 year

• Overall Survival（OS）

  1 year

• Early mortality rate

  3 months

Other Outcomes (2)

• Changes in lymphocyte subsets (NK cells, T cells) before and after treatment

  At the time of enrollment and at 1 month

• Correlation between human leukocyte antigen (HLA) matching and the concentration of lymphocyte subsets

  HLA typing and matching will be assessed at the time of enrollment, and the concentration of lymphocyte subsets in peripheral blood will be detected at the time of enrollment and at 1 month

Study Arms (1)

chemotherapy+Unrelated Umbilical Cord Blood microtransplantation

EXPERIMENTAL

Intermittent infusion of HLA mismatched or incompletely matched granulocyte colony-stimulating factor (G-CSF) mobilized peripheral blood stem cells (G-PBSC) during routine chemotherapy without the use of immunosuppressants, abbreviated as "microtransplantation"

Biological: Unrelated Umbilical Cord Blood; Drug: Venetoclax; Drug: Decetabine; Drug: Azacitidine

Interventions

Unrelated Umbilical Cord Blood BIOLOGICAL

The requirements of unrelated umbilical cord blood: recipient-donor HLA match is 0-3/10, same blood type.

chemotherapy+Unrelated Umbilical Cord Blood microtransplantation

Venetoclax DRUG

Patients will be treated with Venetoclax (100 mg on day 1, 200 mg on day 2 and 400 mg on days 3 to 21)

chemotherapy+Unrelated Umbilical Cord Blood microtransplantation

Decetabine DRUG

Patients will be treated with Decetabine (20mg/m\^2/d on days 1 to 5) or Azacitidine (75mg/m\^2/d on days 1 to 7)

chemotherapy+Unrelated Umbilical Cord Blood microtransplantation

Azacitidine DRUG

Patients will be treated with Decetabine (20mg/m\^2/d on days 1 to 5) or Azacitidine (75mg/m\^2/d on days 1 to 7)

chemotherapy+Unrelated Umbilical Cord Blood microtransplantation

Eligibility Criteria

• Age: 14 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients diagnosed with MDS with WHO criteria through bone marrow morphology, histochemistry, immunophenotyping, pathological testing, etc. Patients can be newly diagnosed, recurrent or unresponsive, with International prognostic scoring system (IPSS-R) score\> 3.5
• Patients aged 14-80, gender and race are not limited;
• Karnofsky score ≥ 60%, Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;
• Expected survival time ≥ 3 months;
• The examination results meet the following requirements:
• ALT and AST ≤ 3 × Upper limit of normal value (ULN); Total bilirubin ≤ 3 × ULN; Creatinine ≤ 2 × ULN or creatinine clearance rate ≥ 40mL/min; The left ventricular ejection fraction (LVEF) measured by echocardiography or multigated acquisition (MUGA) scanning is within the normal range (\>50%);
• The recipient and selected donor should be 0-3/10 HLA-matched with same blood type;
• Patients who voluntarily participate in this clinical study and have signed an informed consent.

You may not qualify if:

• Patients who have suffered from malignant tumors;
• Patients have suffered from hematopoietic failure after chemotherapy, and have undergone ineffective blood transfusion with unknown cause;
• Patients who have undergone Class II or above surgery within 4 weeks prior to enrollment;
• Suffering from life-threatening diseases other than MDS;
• Allergic to the drugs in the research;
• Patient with severe cardiac insufficiency, including uncontrolled or symptomatic arrhythmia, congestive heart failure, myocardial infarction within 6 months, or any grade 3 (moderate) or grade 4 (severe) heart disease;
• Patients with test positive for HIV, HCV or HBV;
• Stroke or intracranial hemorrhage occurred within 6 months prior to enrollment;
• Warfarin or vitamin K antagonists (such as phenprocoumarin) is necessary for anticoagulation;
• Patients with mental illnesses or cognitive impairments;
• Patients have participated within the month prior to enrollment or patients are currently participating in other clinical trials;

Sponsors & Collaborators

• Anhui Provincial Hospital: lead

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China

Hefei, Anhui, 230001, China

RECRUITING

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

venetoclax; Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

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

Study Officials

• Xiaoyu Zhu, MD

  The First Affiliated Hospital of University of Science and Technology of China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoyu Zhu, MD

CONTACT

15255456091; xiaoyuz@ustc.edu.cn

Study Design

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

Study Record Dates

• First Submitted: October 19, 2023
• First Posted: October 31, 2023
• Study Start: October 27, 2023
• Primary Completion: February 1, 2024
• Study Completion: February 1, 2026
• Last Updated: October 31, 2023

Record last verified: 2023-10

Locations

CN (1)