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

F

First Affiliated Hospital of Guangxi Medical University

Status and phase

Completed
Phase 4

Conditions

Thalassemia Major

Treatments

Drug: Busulfan
Drug: Cyclophosphamide
Drug: Tacrolimus
Drug: Mycophenolate mofetil
Drug: Fludarabine
Drug: Methotrexate
Drug: Thymoglobulin
Drug: cyclosporine A

Study type

Interventional

Funder types

Other

Identifiers

NCT04009525
GX-HSCT-MT 2019

Details and patient eligibility

About

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.

Full 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.

Enrollment

823 patients

Sex

All

Ages

2 to 20 years old

Volunteers

No Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

823 participants in 3 patient groups

MSD-HSCT
Experimental group
Description:
matched sibling donors hematopoietic stem cell transplantation
Treatment:
Drug: cyclosporine A
Drug: Thymoglobulin
Drug: Methotrexate
Drug: Fludarabine
Drug: Mycophenolate mofetil
Drug: Cyclophosphamide
Drug: Busulfan
URD-HSCT
Experimental group
Description:
unrelated donor hematopoietic stem cell transplantation
Treatment:
Drug: Thymoglobulin
Drug: Methotrexate
Drug: Fludarabine
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Drug: Cyclophosphamide
Drug: Busulfan
haplo-HSCT
Experimental group
Description:
haplo-identical hematopoietic stem cell transplantation
Treatment:
Drug: Thymoglobulin
Drug: Methotrexate
Drug: Fludarabine
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Drug: Cyclophosphamide
Drug: Busulfan

Trial contacts and locations

1

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Central trial contact

Qiaochuan Li, MD; Yongrong Lai, MD

Data sourced from clinicaltrials.gov

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