Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing Graft-versus-host Disease After Haploidentical/matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia

October 24, 2024 updated by: Rongrong Liu

Evaluation of the Clinical Efficacy and Safety of Recombinant Humanized Anti-CD25 Monoclonal Antibody in Preventing Graft-versus-host Disease After Haploidentical / Matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia: a Prospective, Multicenter, Open-label, Randomized Controlled Clinical Trial

Graft-versus-host disease (GVHD) is a major factor affecting the efficacy and quality of life of alternative donor transplantation in thalassemia major (TM), severely limiting the clinical application of alternative donor transplantation in TM.The purpose of this clinical trial is to evaluate whether recombinant humanized anti-CD25 monoclonal antibody is effective in preventing GVHD and its safety after haploidentical/matched unrelated donor hematopoietic stem cell transplantation. The main questions it aims to answer are:

  • Does recombinant humanized anti-CD25 monoclonal antibody reduce the incidence of GVHD disease after haploidentical/matched unrelated donor hematopoietic stem cell transplantation?
  • What medical problems will participants experience when using the recombinant humanized anti-CD25 monoclonal antibody? What is the quality of life after 2 years follow-up? In this clinical trail, participants will be randomly assigned to the intervention group or the control group by researchers in a 2:1 ratio. The intervention group will be given recombinant humanized anti-CD25 monoclonal antibody (1mg/Kg) combined with the standard GVHD prophylaxis after transplantation, while the control group will only receive the standard GVHD prophylaxis. The incidence of GVHD after transplantation in the two groups will be observed. The main evaluation is the clinical efficacy of recombinant humanized anti-CD25 monoclonal antibody in preventing aGVHD.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

396

Phase

  • Phase 2

Contacts and Locations

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

Study Contact

Study Locations

  • China
    • Guangdong
      • Maoming, Guangdong, China, 525000
        • Recruiting
        • Maoming People's Hospital
        • Contact:
          • Xiong Zhang
    • Guangxi
      • Liuzhou, Guangxi, China, 545007
        • Recruiting
        • Liuzhou Workers' Hospital
        • Contact:
      • Nanning, Guangxi, China, 530000
        • Recruiting
        • The First Affiliated Hospital of Guangxi Medical University
        • Contact:
      • Yulin, Guangxi, China, 537000
        • Recruiting
        • Yulin Red Cross Hospital
        • Contact:
          • Hua Zhang
    • Hainan
      • Haikou, Hainan, China, 570000
        • Recruiting
        • Hainan Provincial People's Hospital
        • Contact:
          • Guyun Wang

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

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • patients with transfusion-dependent thalassemia;
  • patients who are planning to receive matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) or HLA haploidentical donor hematopoietic stem cell transplantation (HID-HSCT);
  • physical condition score (Lansky/Karnofsky score) ≥ 70%;
  • patients (or legal guardians) voluntarily participate in the study and sign the informed consent form

Exclusion Criteria:

  • patients with HLA-matched hematopoietic stem cell donors and willing to receive HLA-matched hematopoietic stem cell transplantation;
  • patients with known infectious diseases such as hepatitis B, hepatitis C, AIDS, syphilis, human T-lymphotropic virus, etc.;
  • patients with serious active bacterial, viral, fungal, malaria or parasitic infections;
  • patients with autoimmune deficiency diseases;
  • patients with a history of malignant tumors or current malignant tumors;
  • patients with important organ diseases or abnormal laboratory tests, including but not limited to: 1) patients with cirrhosis, liver fibrosis or active hepatitis, and/or abnormal liver function tests (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥2.5×ULN; alkaline phosphatase ≥2.5×ULN); 2) patients with heart disease, or left ventricular ejection fraction (LVEF) <60%, or severe iron deposition in the heart; 3) kidney disease, or blood creatinine ≥1.5×ULN with creatinine clearance <30% of normal level; 4) patients with endocrine dysfunction;
  • patients with uncorrected bleeding disease;
  • patients with severe mental illness (such as severe depression, schizophrenia, etc.) or cognitive dysfunction (dementia, delirium, etc.), which are unable to cooperate with the study;
  • peripheral blood white blood cell (WBC) count <3×10^9/L or platelet count <100×10^9/L;
  • patients having received thalidomide treatment within the past 3 months;
  • patients having received any type of gene and/or cell therapy in the past;
  • patients with severe allergies;
  • female patients who are pregnant, breastfeeding, or planning to become pregnant within 1 year of participating in this trial;
  • patients who are participating in other clinical trials;
  • other situations that are not suitable for participation in this clinical trial as assessed by the investigator.

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: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: intervention group
The intervention group will receive 4 doses of recombinant humanized anti-CD25 monoclonal antibody (Sunshine Guojian Pharmaceutical(Shanghai) Co.,Ltd.) on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg.
Drug: recombinant humanized anti-CD25 monoclonal antibody
The intervention group received 4 doses of recombinant humanized anti-CD25 monoclonal antibody on days +7, +14, +28, and +42 after transplantation, with a recommended dose of 1 mg/kg.
No Intervention: control group
The control group will receive no treatment at the same time points.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Grade III-IV aGVHD
Time Frame: within 100 days after HID-HSCT/MUD-HSCT
the incidence of grade III-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
within 100 days after HID-HSCT/MUD-HSCT

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Grade II-IV aGVHD
Time Frame: within 100 days after HID-HSCT/MUD-HSCT
the incidence of grade II-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
within 100 days after HID-HSCT/MUD-HSCT
Chronic graft-versus-host disease (cGVHD)
Time Frame: 2 years
The incidence of cGVHD after HID-HSCT/MUD-HSCT was compared between the intervention group and the control group.
2 years
Overall survival (OS)
Time Frame: 2 years
Comparison of 2-year OS between the intervention group and the control group.
2 years
Thalassemia-free survival (TFS)
Time Frame: 2 years
comparison of the 2-year TFS between the intervention group and the control group.
2 years
Transplantation-related mortality (TRM)
Time Frame: 2 years
comparison of the 2-year TRM between the intervention group and the control group.
2 years
Transplantation-related complications
Time Frame: 2 years
comparison of the 2-year transplantation-related complications between the intervention group and the control group.
2 years
Infection
Time Frame: 2 years
comparison of the infection during the transplantation between the intervention group and the control group.
2 years
Immune reconstitution
Time Frame: 2 years
comparison of the immune reconstitution after transplantation between the intervention group and the control group.
2 years
Adverse events
Time Frame: 2 years
comparison of the occurrence of adverse events between the intervention group and the control group.
2 years
Quality of life
Time Frame: 2 years

comparison of the quality of life between the intervention group and the control group by using the Pediatric Quality of Life Inventory Version 4.0 (PedsQL4.0) scale. The scales are comprissed of parallel self-report and parent proxy-report formats:

  1. Self-report includes ages 8 to 12, 13 to 18, and 18 to 25, with a total of 23 items.
  2. Parent proxy-report assesses parent's perceptions of their child's health-related quality of life. Parent proxy-report (ages 5 to 7) has 23 items, and Parent proxy-report (ages 2 to 4) has 21 items.

A 5-point response scale is utilized across self-report for ages and parent proxy-report (0 = never a problem; 1 = almost never a problem; 2 = sometimes a problem; 3 = often a problem; 4 = almost always a problem). Items are reverse-scored and linearly transformed to a 0 to 100 scale (0 = 100, 1 = 75, 2 = 50, 3 =25, 4 = 0), so that higher scores indicate better health-related quality of life.
2 years

Collaborators and Investigators

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

Sponsor

Rongrong Liu

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)

October 1, 2024

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

September 30, 2027

Study Registration Dates

First Submitted

October 21, 2024

First Submitted That Met QC Criteria

October 23, 2024

First Posted (Actual)

October 24, 2024

Study Record Updates

Last Update Posted (Actual)

October 28, 2024

Last Update Submitted That Met QC Criteria

October 24, 2024

Last Verified

October 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2024-K387-01

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

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.

Subscribe