Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

Overview

The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Sequential Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: May 31, 2022

Detailed Description

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.

Interventions

  • Drug: Busulfan
    • Busulfan(4 mg/kg/day,4 days)
  • Drug: Cyclophosphamide
    • Cyclophosphamide(50 mg/kg/day,4 days)
  • Drug: Fludarabine
    • Fludarabine(50 mg/m2/day,3 days)
  • Drug: Thymoglobulin
    • Thymoglobulin(2.5 mg/kg/day,4 days)
  • Drug: cyclosporine A
    • cyclosporine A
  • Drug: Mycophenolate mofetil
    • Mycophenolate mofetil(0.25g/day)
  • Drug: Tacrolimus
    • Tacrolimus
  • Drug: Methotrexate
    • Methotrexate
  • Drug: Basiliximab
    • Basiliximab
  • Drug: Ruxolitinib
    • Ruxolitinib

Arms, Groups and Cohorts

  • Experimental: MSD-HSCT
    • matched sibling donors hematopoietic stem cell transplantation
  • Experimental: URD-HSCT
    • unrelated donor hematopoietic stem cell transplantation
  • Experimental: haplo-HSCT
    • haplo-identical hematopoietic stem cell transplantation

Clinical Trial Outcome Measures

Primary Measures

  • Overall survival
    • Time Frame: 2 years
    • 2-years overall survival
  • Thalassemia-free survival
    • Time Frame: 2 years
    • 2-years thalassemia-free survival

Secondary Measures

  • Engraftment
    • Time Frame: 30 days
    • Myeloid engraftment at day +30
  • Transplant Related Mortality
    • Time Frame: 1 year
    • Transplant-related mortality by 1 year
  • Cumulative Incidence of acute Graft Versus Host Disease
    • Time Frame: 180 days
    • Acute graft versus host disease at day +180
  • Cumulative Incidence of chronic Graft Versus Host Disease
    • Time Frame: 2 years
    • Chronic graft versus host disease by 2 years
  • Cumulative Incidence of Infectious Complications
    • Time Frame: 2 years
    • Cumulative incidence of bacterial, fungal and viral infections by 2 years

Participating in This Clinical Trial

Inclusion Criteria

1. Diagnosed with thalassemia major. 2. Indication of hematopoietic stem cell transplantation. 3. A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function. Exclusion Criteria:

1. Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria; 2. Uncontrolled bacterial, viral or fungal infections; 3. Any other restriction for transplantation.

Gender Eligibility: All

Minimum Age: 2 Years

Maximum Age: 20 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • First Affiliated Hospital of Guangxi Medical University
  • Collaborator
    • Peking University People’s Hospital
  • Provider of Information About this Clinical Study
    • Principal Investigator: Yongrong Lai, Director of the Hematology Department – First Affiliated Hospital of Guangxi Medical University
  • Overall Official(s)
    • Yongrong Lai, MD, Principal Investigator, First Affiliated Hospital of Guangxi Medical University
  • Overall Contact(s)
    • Yongrong Lai, MD, +86(0771)5356510, laiyongrong@hotmail.com

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