NCT04009525

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 823 (actual)
• Sponsor: First Affiliated Hospital of Guangxi Medical University · Academic / Other
• Sex: All
• Age: 2 Years – 20 Years
• Healthy volunteers: Not accepted

Summary

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.

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.

Conditions

• Thalassemia Major

Interventions

Type	Name	Description
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

Timeline

• Start date: 2019-07-05
• Primary completion: 2023-07-31
• Completion: 2023-10-31
• First posted: 2019-07-05
• Last updated: 2024-08-09

Locations

1 site across 1 country: China