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

Thalassemia Major Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04009525
• Other study ID #: GX-HSCT-MT 2019
• Status: Recruiting
• Phase: Phase 4
• Start date: June 1, 2019
• Est. completion date: May 31, 2023

Study information

• Verified date: July 2019
• Source: First Affiliated Hospital of Guangxi Medical University
• Contact: Yongrong Lai, MD
• Phone: +86(0771)5356510
• Email: laiyongrong[@]hotmail.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 800
• Est. completion date: May 31, 2023
• Est. primary completion date: May 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 20 Years

Eligibility

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.

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Thalassemia Major

Intervention

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

Locations

Country	Name	City	State
China	First Affiliated Hospital of Guangxi Medical University	Nanning	Guangxi

Sponsors (9)

Lead Sponsor	Collaborator
First Affiliated Hospital of Guangxi Medical University	Fourth Affiliated Hospital of Guangxi Medical University, Hainan General Hospital, Liuzhou General Hospital, Peking University People's Hospital, Ruijin Hospital, The 923th Hospital of People's Liberation Army, The Affiliated Hospital Of Guizhou Medical University, The First People's Hospital of Yunnan

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall survival	2-years overall survival	2 years
Primary	Thalassemia-free survival	2-years thalassemia-free survival	2 years
Secondary	Engraftment	Myeloid engraftment at day +30	30 days
Secondary	Transplant Related Mortality	Transplant-related mortality by 1 year	1 year
Secondary	Cumulative Incidence of acute Graft Versus Host Disease	Acute graft versus host disease at day +180	180 days
Secondary	Cumulative Incidence of chronic Graft Versus Host Disease	Chronic graft versus host disease by 2 years	2 years
Secondary	Cumulative Incidence of Infectious Complications	Cumulative incidence of bacterial, fungal and viral infections by 2 years	2 years