Study of Human Umbilical Cord-derived Mesenchymal Stem Cells for Treatment of Refractory Immune Thrombocytopenia

February 20, 2025 updated by: Institute of Hematology & Blood Diseases Hospital, China

Primary Objective: To evaluate the safety and efficacy of human umbilical cord-derived mesenchymal stem cells（hUC-MSCs） to treat refractory immune thrombocytopenia（ITP）.

Secondary Objective: To observe the changes of immune function in refractory ITP patients with human umbilical cord-derived mesenchymal stem cells（hUC-MSCs） after infusion, and to explore and reveal the mechanism of hUC-MSCs in treating ITP.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Human umbilical cord (hUC)-derived mesenchymal stem cells (MSCs) have been shown to have marked therapeutic effects in a number of inflammatory diseases and autoimmune diseases in humans based on their potential for immunosuppression and their low immunogenicity. Currently, no more data is available on the safety and effectiveness of hUC-MSCs to treat immune thrombocytopenia patients.

This is a single-arm study to evaluate the safety and efficacy of hUC-MSCs to treat refractory immune thrombocytopenia（ITP）. In addition, it is the objective of this study to observe the changes of immune function in refractory ITP patients after hUC-MSCs infusion, and to explore and reveal the mechanism of hUC-MSCs in treating ITP.

The investigator will assess the changes of the platelet counts after hUC-MSCs infusion from week 1 to week 28, and observe incidence of adverse events during and after hUC-MSCs infusion.The investigator will complete virus detection( including HBV, HCV, HIV, Syphilis, etc) at week 4 and week 16 after hUC-MSCs infusion.

The dose of hUC-MSCs will be successively divided into three increasing dose(group A: hUC-MSCs 0.5×10^6/kg, weekly for 4 weeks, 3 patients; group B: hUC-MSCs 1.0×10^6/kg, weekly for 4 weeks, 3 patients; hUC-MSCs 2.0×10^6/kg, weekly for 4 weeks, 3 patients) with 3 patients in each group according to the dose.

The principle of increasing dose will be carried out successively from low dose to high dose group. According to the results of the safety and efficacy data from these 9 patients, the investigator will determine one of the doses and expand the sample size to 6 cases.

The investigator will observe the concentration of hUC-MSCs in peripheral blood from female patients after the first hUC-MSCs infusion at 10 time points, including 30 minutes before hUC-MSCs infusion, 30 minutes, 60 minutes, 2 hours, 4 hours, 8 hours, 16 hours, 24 hours,48 hours and 96 hours after the first hUC-MSCs infusion.

The investigator will detect antibody production of hUC-MSCs in peripheral blood from the first 9 patients at 2 time points, including 30 minutes before the first hUC-MSCs infusion and 48 hours after the last hUC-MSCs infusion.

The investigator will observe the changes of immune function in refractory ITP patients after hUC-MSCs infusion at 7 time points, including one day before hUC-MSCs infusion, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16weeks and 28 weeks after hUC-MSCs infusion.

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Yunfei Chen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Aged 18 to 60 years old, male or female;
  • Conform to the diagnostic criteria of immune Thrombocytopenia (ITP);
  • Three months after splenectomy;
  • The first-line treatment drugs such as human immunoglobulin, glucocorticoid, and the second-line treatment of thrombopoietin drugs and rituximab were invalid, or there was no response or recurrence after splenectomy;
  • Diagnosis of ITP>6 months；
  • More than 3 months after rituximab treatment；
  • Platelet counts <30 ×10^9/L, and bleeding tendency;
  • People who are willing to sign the informed consent voluntarily and follow the research program.
  • Subject is practicing an acceptable method of contraception. Women of childbearing potential must have a negative serum pregnancy test in the whole study;

Exclusion Criteria:

  • ECOG score standard >2；
  • Secondary thrombocytopenic purpura;
  • Patients with poor compliance;
  • Positive serology for HIV, hepatitis B virus (HBV), hepatitis C virus (HCV), and/or hepatitis D virus (HDV), Syphilis; Positive for Epstein-Barr Virus DNA, Cytomegalovirus DNA;
  • Pregnancy or lactation period;
  • History of thrombosis;
  • The serum chemistry results exceed the upper laboratory normal range by more than 20%, such as ALT, AST, TBIL, BUN, Cre etc;
  • Pre-existing cardiac disease, including congestive heart failure of New York Heart Association [NYHA] Grade III/IV, arrhythmia requiring treatment or myocardial infarction within the last 6 months. No arrhythmia known to increase the risk of thrombotic events (e.g. atrial fibrillation), or patients with a QT >450msec or QTc > 480 for patients with a Bundle Branch Block;
  • History of solid organ or bone marrow transplant;
  • Researchers believe that patients should not participate in the test of any other condition.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 15 refractory ITP patients
15 enrolled refractory ITP patients will be picked up to infuse hUC-MSCs at the indicated dose.
Other: Human umbilical cord-derived mesenchymal stem cells (hUC-MSCs)
This is a single-arm study to evaluate the safety and efficacy of hUC-MSCs to treat refractory immune thrombocytopenia. The dose of hUC-MSCs will be successively divided into three increasing dose(group A: hUC-MSCs 0.5×10^6/kg, weekly for 4 weeks, 3 patients; group B: hUC-MSCs 1.0×10^6/kg, weekly for 4 weeks, 3 patients; hUC-MSCs 2.0×10^6/kg, weekly for 4 weeks, 3 patients) with 3 patients in each group according to the dose. The principle of increasing dose will be carried out successively from low dose to high dose group. According to the results of the safety and efficacy data from these 9 patients, the investigator will determine one of the doses and expand the sample size to 6 cases.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes of the platelet counts after hUC-MSCs infusion
Time Frame: 28 weeks
The investigator will assess the changes of the platelet counts after hUC-MSCs infusion from week 1 to week 28.
28 weeks
Incidence of adverse events after hUC-MSCs infusion
Time Frame: 4 weeks
The investigator will observe incidence of adverse events during and after hUC-MSCs infusion, including fever, thrombosis, diarrhea, skin rash and so on.
4 weeks
Changes in virus safety indicators after hUC-MSCs infusion
Time Frame: 16 weeks
The investigator will complete virus detection( including HBV, HCV, HIV, Syphilis, EB, CMV, etc) at week 4 and week 16 after hUC-MSCs infusion.
16 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes of concentration of hUC-MSCs in peripheral blood
Time Frame: 96 hours
The investigator will observe the concentration of hUC-MSCs in peripheral blood from female patients after the first hUC-MSCs infusion at 10 time points, including 30 minutes before hUC-MSCs infusion, 30 minutes, 60 minutes, 2 hours, 4 hours, 8 hours, 16 hours, 24 hours,48 hours and 96 hours after the first hUC-MSCs infusion.
96 hours
Changes of antibody production of hUC-MSCs in peripheral blood
Time Frame: 24 days
The investigator will detect antibody production of hUC-MSCs in peripheral blood from the first 9 patients at 2 time points, including 30 minutes before the first hUC-MSCs infusion and 48 hours after the last hUC-MSCs infusion.
24 days
Changes of immune function in refractory ITP patients after hUC-MSCs infusion
Time Frame: 28 weeks
The investigator will observe the changes of immune function in refractory ITP patients after hUC-MSCs infusion at 7 time points, including one day before the first hUC-MSCs infusion, 2 weeks, 4 weeks, 8 weeks, 12 weeks, 16weeks and 28 weeks after the first hUC-MSCs infusion. The changes of immune function will include the changes of the percentage of Th cell subsets， regulatory T cells (Treg)，supressor T cells（Ts）, activation and proliferation of B and T lymphocyte, apoptosis of platelets by cytotoxic T cells(CTLs) and function of dendritic cells in peripheral blood mononuclear cells（PBMCs）at the 7 time points, and to compare with the healthy controls. The investigator also will assess the changes of cytokines in the cell culture supernatants and plasma at the 7 time points, and to compare with the healthy controls.
28 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Investigators

  • Principal Investigator: lei zhang, MD, Chinese Academy of Medical Science and Blood Disease Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 21, 2019

Primary Completion (Actual)

February 28, 2023

Study Completion (Actual)

June 30, 2023

Study Registration Dates

First Submitted

July 7, 2019

First Submitted That Met QC Criteria

July 7, 2019

First Posted (Actual)

July 10, 2019

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

February 20, 2025

Last Verified

June 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SC2019002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.

IPD Sharing Time Frame

From 12 months 36 months after study completion.

IPD Sharing Access Criteria

Upon request to PI.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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