Efficacy and Safety of UC-MSCs for the Treatment of Steroid-resistant aGVHD Following Allo-HSCT

Sponsor
Peking University People's Hospital (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT04738981
Collaborator
Southern Medical University, China (Other), Third Military Medical University (Other), Sun Yat-sen University (Other)
130
1
2
10
13.1

Study Details

Study Description

Brief Summary

Randomized, open-label, multicenter study to investigate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSC) for the treatment of steroid-resistant acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a view to establishing an effective treatment protocol for steroid-resistant aGVHD.

Condition or Disease Intervention/Treatment Phase
Phase 3

Detailed Description

A total of 130 patients with steroid-resistant aGVHD after allo-HSCT are enrolled in this multicenter, randomized, controlled trial. Patients were randomized to UC-MSC + anti-CD25 monoclonal antibodies (mAb) group and anti-CD25 mAb group. The complete response (CR) rate after 4 weeks of treatment in the two groups will be compared. Adverse events are also recorded throughout the study.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
130 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Efficacy and Safety of UC-MSCS for the Treatment of Steroid-resistant aGVHD Following Allo-HSCT: A Multicenter, Randomized, Open-label Trial
Anticipated Study Start Date :
Feb 1, 2021
Anticipated Primary Completion Date :
Dec 1, 2021
Anticipated Study Completion Date :
Dec 1, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: UC-MSC and anti-CD25 mAb

UC-MSC, iv, 1×10^6 cells/kg, once a week, for 4 weeks. Anti-CD25 mAb, iv, 20mg,twice in first week and once a week thereafter, 4 weeks. Other treatment would replace it according to clinical experience if aGVHD continue to progress within 3 weeks of treatment or patients are lack of response after 4 weeks of treatment. The treatment would be repeated in another 4 weeks if patients receive partial response after the first 4 weeks of treatment.

Drug: UC-MSC
UC-MSC, iv, 1×10^6 cells/kg, once a week, for 4 weeks.
Other Names:
  • Umbilical cord-derived mesenchymal stem cells
  • Drug: Anti-CD25 mAb
    Anti-CD25 mAb, iv, 20mg,twice in first week and once a week thereafter, for 4 weeks.
    Other Names:
  • Basiliximab
  • Active Comparator: Anti-CD25 mAb

    Anti-CD25 mAb, iv, 20mg,twice in first week and once a week thereafter, for 4 weeks. Other treatment would replace it according to clinical experience if aGVHD continue to progress within 3 weeks of treatment or patients are lack of response after 4 weeks of treatment. The treatment would be repeated in another 4 weeks if patients receive partial response after first 4 weeks of treatment.

    Drug: Anti-CD25 mAb
    Anti-CD25 mAb, iv, 20mg,twice in first week and once a week thereafter, for 4 weeks.
    Other Names:
  • Basiliximab
  • Outcome Measures

    Primary Outcome Measures

    1. Rate of complete remission [4 weeks after treatment]

      Complete remission of aGVHD related symptoms and indicators

    Secondary Outcome Measures

    1. Overall survival [At the end of Week 4 / 8 / 12 / 24 / 52.]

      The time from randomization to death for any reason ( the last follow-up time for missing patients; end date of follow-up for patients who are live at the end of the study).

    2. Rate of partial remission [4 weeks after treatment]

      improvement of aGVHD staging in one or more organs without progression in other organs

    3. Infusion toxicity [From the beginning of to four hours after every infusion of UC-MSC]

      Acute toxicity responses include impaired function of heart, kidney and liver

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years to 70 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • 1.Confirmed diagnosed steroid-resistant aGVHD after allo-HSCT. 2. Neutrophile granulocyte (ANC) ≥ 0.5 x 10^9/L. 3. Creatinine below 2 times normal upper limit . 4. Willing and able to sign written informed consent.
    Exclusion Criteria:
      1. Untoleratation of the treatment. 2. Primary disease are not effectively controlled or are progressing. 3. Active infection (Bacteria, fungi, viruses ). 4. Combined with dysfunction of multiple organ. 5. Female patients who are nursing or pregnant, or who contemplate pregnancy during the study period.
    1. Patients who are deemed unsuitable for the study by the investigator.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Peking University Insititute of Hematology, Peking University People's Hospital Beijing Beijing China 100010

    Sponsors and Collaborators

    • Peking University People's Hospital
    • Southern Medical University, China
    • Third Military Medical University
    • Sun Yat-sen University

    Investigators

    • Principal Investigator: Xiaohui Zhang, doctor, Peking University People's Hospital, Peking University Insititute of Hematology

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Xiao Hui Zhang, Vice president of Peking Univeristy Institute of Hematology, Peking University People's Hospital
    ClinicalTrials.gov Identifier:
    NCT04738981
    Other Study ID Numbers:
    • GVHD-PKU2021
    First Posted:
    Feb 4, 2021
    Last Update Posted:
    Feb 4, 2021
    Last Verified:
    Feb 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Feb 4, 2021