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

Enrollment

Location

Arms

Anticipated Duration (Months)

13.1

Patients Per Site Per Month

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
Graft Vs Host Disease	Drug: UC-MSC Drug: Anti-CD25 mAb	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

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

Rate of complete remission [4 weeks after treatment]
Complete remission of aGVHD related symptoms and indicators

Secondary Outcome Measures

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).
Rate of partial remission [4 weeks after treatment]
improvement of aGVHD staging in one or more organs without progression in other organs
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:

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.

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:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Graft vs Host Disease

Basiliximab

Study Results

No Results Posted as of Feb 4, 2021