Trial | NCT04009525  Report issue

Official Title

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

  • Phase

    Phase 4

  • Study Type

    Interventional

  • Status

    Unknown status

  • Study Participants

    800
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.
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.
Study Started
Jun 01
2019
Primary Completion
May 31
2022
Anticipated
Study Completion
May 31
2023
Anticipated
Last Update
Jul 05
2019

Drug Busulfan

Busulfan（4 mg/kg/day,4 days）

  • Other names: Bu

Drug Cyclophosphamide

Cyclophosphamide（50 mg/kg/day,4 days）

  • Other names: Cy

Drug Fludarabine

Fludarabine（50 mg/m2/day,3 days）

  • Other names: Flu

Drug Thymoglobulin

Thymoglobulin（2.5 mg/kg/day,4 days）

  • Other names: ATG

Drug cyclosporine A

cyclosporine A

  • Other names: CsA

Drug Mycophenolate mofetil

Mycophenolate mofetil（0.25g/day）

  • Other names: MMF

Drug Tacrolimus

Tacrolimus

  • Other names: FK506

Drug Methotrexate

Methotrexate

  • Other names: MTX

Drug Basiliximab

Basiliximab

  • Other names: Simulect

Drug Ruxolitinib

Ruxolitinib

MSD-HSCT Experimental

matched sibling donors hematopoietic stem cell transplantation

URD-HSCT Experimental

unrelated donor hematopoietic stem cell transplantation

haplo-HSCT Experimental

haplo-identical hematopoietic stem cell transplantation

Criteria 

Inclusion Criteria:

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

Exclusion Criteria:

Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;
Uncontrolled bacterial, viral or fungal infections;
Any other restriction for transplantation.
No Results Posted