A Randomized Controlled, Open, Single-center Clinical Study Evaluating the Efficacy and Safety of Umbilical Cord Blood Mononuclear Cells in Patients with Refractory Immune Effector Cell-related Hemocytopenia

The purpose of this study was to evaluate the safety and efficacy of umbilical cord blood mononuclear cell in the treatment of refractory immune effector cell-related hemocytopenia by observing the efficacy related factors and adverse reactions.

Study Overview

• Status: Not yet recruiting
• Conditions: Refractory Immune Effector Cell-related Hemocytopenia
• Intervention / Treatment: Combination product: Conventional hematopoietic recovery therapy; Biological: Umbilical cord blood mononuclear cells

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Kai Hu; Phone Number: +86 150 1039 0336; Email: huk@gobroadhealthcare.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 102200
      • Beijing GoBroad Hospital
      • Contact: Kai Hu; Phone Number: +86 150 1039 0336; Email: huk@gobroadhealthcare.com

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Age ≥18 years old (including boundary values), gender is not limited
2. ECOG score 0-2 points
3. Expected survival ≥6 months
4. Patients who have received immune effector cell therapy (marketed CAR-T cell therapy or dual antibody therapy) (no target), have grade 3-4 hemocytopenia after treatment, and do not recover to grade 2 within 3 weeks after conventional treatment. Defined as follows:

1) The neutrophils again decreased to grade ≥3 within 1 week after G-CSF discontinuation, or 2) Unable to detach from red blood cell or platelet transfusion (infusion time less than one week requires re-infusion) (5) Understand the research protocol and sign the informed consent voluntarily

Exclusion Criteria:

1. Patients who intend to undergo autologous hematopoietic stem cell transfusion or have undergone autologous hematopoietic stem cell transfusion
2. Significant lack of compliance to complete the study plan (such as suffering from uncontrolled mental illness, etc.)
3. Allergy or known allergy to any drug active ingredients, excipients, and blood products or preparations included in this study
4. Pregnancy, breastfeeding, planning pregnancy, or unwillingness to use effective contraception as required by research
5. There are other conditions that researchers believe are not suitable for inclusion

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Conventional hematopoietic recovery therapy; Conventional hematopoietic recovery therapy including but not limited to G-CSF, TPO, TPO receptor agonists, transfusions of red blood cells, platelets, etc.	Combination product: Conventional hematopoietic recovery therapy; Including but not limited to G-CSF, TPO, TPO receptor agonists, transfusions of red blood cells, platelets, etc.
Experimental: Conventional hematopoietic recovery therapy+Umbilical cord blood mononuclear cells; Umbilical cord blood mononuclear cells are obtained from umbilical cord blood by density gradient centrifugation	Combination product: Conventional hematopoietic recovery therapy; Including but not limited to G-CSF, TPO, TPO receptor agonists, transfusions of red blood cells, platelets, etc.; Biological: Umbilical cord blood mononuclear cells; Intravenous infusion of UCB-MNCs (3×10^8/ time, once a week, four times in total)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The recovery time of neutrophils	On the first day after discontinuing G-CSF, the neutrophil count remained above 1.0 × 10^9/L for 7 consecutive days	1 year
The recovery time of platelet	Starting from the second day of the most recent platelet transfusion, the first day of platelet count>50 × 10^9/L lasting for 7 days	1 year
Safety of infusion	Acute allergies or infections related to umbilical cord blood mononuclear cell infusion, as well as shock and other infusion related reactions symptoms including but not limited to chills, fever, CRP, PCT, etc	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Recovery of hemoglobin levels after infusion	Hemoglobin content in blood routine examination	1 year
Immune function reconstruction indicators at 1, 3, 6, and 12 months after transfusion	Including but not limited to CD3, CD4, CD8, CD19, etc. in lymphoid subgroups	1, 3, 6, and 12 months
Overall survival rate	The proportion of participants who survived during the study period	2 and 5 year

Collaborators and Investigators

• Sponsor: Beijing GoBroad Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2024
• Primary Completion (Estimated): June 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: December 8, 2024
• First Submitted That Met QC Criteria: December 8, 2024
• First Posted (Estimated): December 11, 2024

Study Record Updates

• Last Update Posted (Estimated): December 11, 2024
• Last Update Submitted That Met QC Criteria: December 8, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: UCB-MNCs-2024

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No