Using a monoclonal antibody to prevent graft-versus-host disease in thalassemia patients after stem cell transplantation

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

This study is testing if a new antibody can help prevent graft-versus-host disease in thalassemia patients who are receiving stem cell transplants.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 396 (estimated)
• Ages: 3 Years to 20 Years
• Sex: All
• Sponsor: First Affiliated Hospital of Guangxi Medical University (other)
• Locations: 5 sites (Maoming, Guangdong and 4 other locations)
• Trial ID: NCT06657391 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the effectiveness of a recombinant humanized anti-CD25 monoclonal antibody in preventing graft-versus-host disease (GVHD) in patients with transfusion-dependent thalassemia undergoing haploidentical or matched unrelated donor hematopoietic stem cell transplantation. Participants will be randomly assigned to receive either the monoclonal antibody or a control treatment. The study aims to determine the incidence of GVHD and assess the safety and quality of life of participants over a two-year follow-up period.

Who should consider this trial

Good fit: Ideal candidates are patients with transfusion-dependent thalassemia who are planning to receive haploidentical or matched unrelated donor stem cell transplants.

Not a fit: Patients with HLA-matched donors or those with active infections or serious health conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the incidence of GVHD, improving outcomes and quality of life for patients undergoing stem cell transplantation.

How similar studies have performed: While this approach is novel in the context of thalassemia, similar monoclonal antibody treatments have shown promise in preventing GVHD in other transplant settings.

Eligibility criteria

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.

Where this trial is running

Maoming, Guangdong and 4 other locations

• Maoming People's Hospital — Maoming, Guangdong, China (RECRUITING)
• Liuzhou Workers' Hospital — Liuzhou, Guangxi, China (RECRUITING)
• The First Affiliated Hospital of Guangxi Medical University — Nanning, Guangxi, China (RECRUITING)
• Yulin Red Cross Hospital — Yulin, Guangxi, China (RECRUITING)
• Hainan Provincial People's Hospital — Haikou, Hainan, China (RECRUITING)

Study contacts

• Study coordinator: Rongrong Liu

Conditions: Transfusion Dependent Thalassemia