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

  • STATUS
    Recruiting
  • End date
    Dec 31, 2022
  • participants needed
    15
  • sponsor
    Institute of Hematology & Blood Diseases Hospital
Updated on 11 July 2021
coagulopathy
platelet count
splenectomy
autoimmune thrombocytopenia

Summary

Primary Objective: To evaluate the safety and efficacy of human umbilical cord-derived mesenchymal stem cellshUC-MSCs to treat refractory immune thrombocytopeniaITP.

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

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 thrombocytopeniaITP. 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 0.510^6/kg, weekly for 4 weeks, 3 patients; group B: hUC-MSCs 1.010^6/kg, weekly for 4 weeks, 3 patients; hUC-MSCs 2.010^6/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.

Details
Condition Thrombocytopenia, cells mesenchymal, Thrombocytopenia and Thrombocytopenia Prevention
Treatment Human umbilical cord-derived mesenchymal stem cells (hUC-MSCs)
Clinical Study IdentifierNCT04014166
SponsorInstitute of Hematology & Blood Diseases Hospital
Last Modified on11 July 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Aged 18 to 60 years old, male or female
Conform to the diagnostic criteria of immune Thrombocytopenia (ITP)
Three months after splenectomy
Platelet counts <30 10^9/L, and bleeding tendency
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
People who are willing to sign the informed consent voluntarily and follow the research program
Diagnosis of ITP>6 months
More than 3 months after rituximab treatment
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 >1
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
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