The Safety and Efficacy of Umbilical Cord Blood Mononuclear Cells in Patients With Secondary Poor Graft Function After Hematopoietic Stem Cell Transplantation

Clinical Study to Explore the Safety and Efficacy of Umbilical Cord Blood Mononuclear Cells in Patients With Secondary Poor Graft Function After Hematopoietic Stem Cell Transplantation

This study is conducted in a prospective, single-center clinical design and is divided into two stages: dose escalation and dose extension. Patients meeting the diagnostic criteria of secondary poor graft function are selected as the study objects. The safety data of umbilical cord blood mononuclear cells in the treatment of secondary poor graft function are obtained through dose escalation stage, and then one dose is selected for dose extension stage to explore the efficacy of umbilical cord blood mononuclear cells in treating secondary poor graft function.

Study Overview

• Status: Recruiting
• Conditions: Poor Graft Function
• Intervention / Treatment: Biological: Dose escalation; Biological: Dose extension

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: erlie EL Jiang; Phone Number: +86-15122538106; Email: jiangerlie@ihcams.ac.cn

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Recruiting
      • Institute of Hematology & Blood Diseases Hospital, China
      • Contact: jiang erlie; Phone Number: +86-15122538106; Email: jiangerlie@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age≥18 years old; gender is not limited.
2. Meet the diagnostic criteria for secondary poor graft function: After 28 days of transplantation, patients who had achieved hematopoietic reestablishment (ANC ≥ 0.5×10^9/L for 3 consecutive days without G-CSF application, PLT ≥ 20×10^9/L for 7 consecutive days without platelet infusion, Hb ≥ 80g/L for 2 consecutive weeks without red blood cell infusion) again developed two or three line cytopenia lasting more than 2 weeks. Bone marrow examination revealed low myelodysplasia, remission of primary disease, complete donor chimeric cells, and no severe graft-versus-host disease (GVHD) or disease recurrence.
3. Eastern Cooperative Oncology Group (ECOG) physical status score ≤ 2 points.
4. Subjects sign informed consent.

Exclusion Criteria:

1. Serious infection not controlled.
2. Active bleeding.
3. Patients with cardiac insufficiency (ejection fraction<50%), or suffering from serious heart disease, including myocardial infarction, cardiac insufficiency, etc.
4. Patients with hepatic and renal insufficiency (total bilirubin>35µmol/L, ALT and AST>2 times of the upper limit of normal; serum creatinine>130µmol/L).
5. Pregnant or lactating women.
6. Concurrent malignant tumors of other organs.
7. Failure to understand or follow the research protocol.
8. Patients participating in other clinical investigations.
9. Other conditions that the investigators consider inappropriate to participate in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Umbilical Cord Blood Mononuclear Cells

Biological: Dose escalation; Three dose groups are preset. The doses are 2.0×10^6/kg/time, 3.5×10^6/kg/time and 5.0×10^6/kg/ time respectively, in accordance with the "3+3" dose escalation principle, and proceed in turn. Each subject receives conventional treatment plus umbilical cord blood mononuclear cells therapy, with 4 consecutive intravenous infusions of umbilical cord blood mononuclear cells at 1 week intervals.; Biological: Dose extension; According to the safety data of dose escalation stage, one dose group is selected for extension. Each subject receives conventional treatment plus umbilical cord blood mononuclear cells therapy, with 4 consecutive intravenous infusions of umbilical cord blood mononuclear cells at 1 week intervals.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety assessment	Incidence of adverse events	From date of the last infusion of umbilical cord blood mononuclear cells until the date of end of follow-up, assessed up to 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The recovery time of platelet	The first day of 3 consecutive days with PLT ≥ 20×10^9/L without platelet transfusion	Two months after the last infusion of umbilical cord blood mononuclear cells
The recovery time of neutrophil	The first day of 3 consecutive days with ANC ≥ 0.5×10^9/L without G-CSF application	Two months after the last infusion of umbilical cord blood mononuclear cells

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Actual): February 14, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): February 1, 2028

Study Registration Dates

• First Submitted: December 27, 2024
• First Submitted That Met QC Criteria: January 21, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 11, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: Umbilical cord blood mononuclear cells; Secondary poor graft function
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Neoplastic Processes; Neoplasm Metastasis
• Other Study ID Numbers: IIT2024098

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No