Completed

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

What is being tested

Busulfan

+ Cyclophosphamide

+ Fludarabine

Drug

Who is being recruted

Anemia+7

+ Anemia, Hemolytic

+ Anemia, Hemolytic, Congenital

From 2 to 20 Years

+6 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 4

Interventional

Study Start: July 2019

See protocol details

Summary

Principal SponsorFirst Affiliated Hospital of Guangxi Medical University

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: July 5, 2019

Actual date on which the first participant was enrolled.

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.

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

NCT04009525

Principal SponsorFirst Affiliated Hospital of Guangxi Medical University

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

823 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 2 to 20 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

AnemiaAnemia, HemolyticAnemia, Hemolytic, CongenitalHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesThalassemiabeta-ThalassemiaGenetic Diseases, Inborn

Criteria

3 inclusion criteria required to participate

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.

3 exclusion criteria prevent from participating

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.

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

3 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

matched sibling donors hematopoietic stem cell transplantation

Group II

Experimental

unrelated donor hematopoietic stem cell transplantation

Group III

Experimental

haplo-identical hematopoietic stem cell transplantation

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Suspended

First Affiliated Hospital of Guangxi Medical University

Nanning, ChinaOpen First Affiliated Hospital of Guangxi Medical University in Google Maps

CompletedOne Study Center