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

Phase 2

Recruiting

• Conditions: Myelodysplastic Syndromes
• Interventions: Biological: Unrelated Umbilical Cord Blood; Drug: Venetoclax; Drug: Decetabine; Drug: Azacitidine

• Registration Number: NCT06109064
• Lead Sponsor: Anhui Provincial Hospital

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2023-10
• Study First Submitted: 2023-10-19
• Study Start: 2023-10-27
• Study First Submitted QC: 2023-10-27
• Last Update Submitted: 2023-10-27
• Study First Posted: 2023-10-31
• Last Update Posted: 2023-10-31
• Primary Completion: 2024-02-01
• Study Completion: 2026-02-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

1. 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

2. Patients aged 14-80, gender and race are not limited;

3. Karnofsky score ≥ 60%, Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2;

4. Expected survival time ≥ 3 months;

5. 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%);

6. The recipient and selected donor should be 0-3/10 HLA-matched with same blood type;

7. Patients who voluntarily participate in this clinical study and have signed an informed consent.

Exclusion Criteria

1. Patients who have suffered from malignant tumors;
2. Patients have suffered from hematopoietic failure after chemotherapy, and have undergone ineffective blood transfusion with unknown cause;
3. Patients who have undergone Class II or above surgery within 4 weeks prior to enrollment;
4. Suffering from life-threatening diseases other than MDS;
5. Allergic to the drugs in the research;
6. 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;
7. Patients with test positive for HIV, HCV or HBV;
8. Stroke or intracranial hemorrhage occurred within 6 months prior to enrollment;
9. Warfarin or vitamin K antagonists (such as phenprocoumarin) is necessary for anticoagulation;
10. Patients with mental illnesses or cognitive impairments;
11. Patients have participated within the month prior to enrollment or patients are currently participating in other clinical trials;
12. There are other conditions that the investigators consider inappropriate for inclusion.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation	Unrelated Umbilical Cord Blood	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"
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation	Decetabine	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"
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation	Venetoclax	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"
chemotherapy+Unrelated Umbilical Cord Blood microtransplantation	Azacitidine	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"

Primary Outcome Measures

Name	Time	Method
Complete response (CR) rate	28±7 days	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.
Hematopoietic recovery time	28±7 days	The time of absolute neutrophil count\>0.5×10\^9/L and platelet count \>30×10\^9/L for 3 consecutive days.

Secondary Outcome Measures

Name	Time	Method
Time to Progression（TTP）	1 year	Time from enrollment to objective progression of disease
Disease Free Survival（DFS）	1 year	From CR to recurrence or death or to the date of last follow-up
Overall Survival（OS）	1 year	From the beginning of treatment to death or to the date of last follow-up
Early mortality rate	3 months	Death within the first 3 months of induction therapy

Trial Locations

Locations (1)

The First Affiliated Hospital of University of Science and Technology of China; 🇨🇳 Hefei, Anhui, China