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NCT Number: NCT04009525

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

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.

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Key information

Conditions

Thalassemia Major Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia beta-Thalassemia

Age range

2 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, 510515, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

busulfan

Drug

Busulfan（4 mg/kg/day,4 days）

Other names: Bu

Cyclophosphamide

Drug

Cyclophosphamide（50 mg/kg/day,4 days）

Other names: Cy

Fludarabine

Drug

Fludarabine（50 mg/m2/day,3 days）

Other names: Flu

Thymoglobulin

Drug

Thymoglobulin（2.5 mg/kg/day,4 days）

Other names: ATG

Cyclosporine A

Drug

cyclosporine A

Other names: CsA

Mycophenolate mofetil

Drug

Mycophenolate mofetil（0.25g/day）

Other names: MMF

Tacrolimus

Drug

Tacrolimus

Other names: FK506

methotrexate

Drug

Methotrexate

Other names: MTX

Primary outcomes

  1. Overall survival

    Time frame: 2 years

    2-years overall survival

  2. Thalassemia-free survival

    Time frame: 2 years

    2-years thalassemia-free survival

Secondary outcomes

  1. Engraftment

    Time frame: 30 days

    Myeloid engraftment at day +30

  2. Transplant Related Mortality

    Time frame: 2 year

    Transplant-related mortality by 2 year

  3. Cumulative Incidence of acute Graft Versus Host Disease

    Time frame: 100 days

    Acute graft versus host disease at day +100

  4. Cumulative Incidence of chronic Graft Versus Host Disease

    Time frame: 2 years

    Chronic graft versus host disease by 2 years

  5. Cumulative Incidence of Infectious Complications

    Time frame: 2 years

    Cumulative incidence of bacterial, fungal and viral infections by 2 years

Sponsors and collaborators

Lead sponsor

First Affiliated Hospital of Guangxi Medical University

Other

Collaborators

  • 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
  • Affiliated Hospital of Youjiang University of Ethnic Medicine
  • Guangxi Autonomous Region People's Hospital
  • Guilin Medical College
  • Hainan People's Hospital
  • LiuZhou People's Hospital
  • Liuzhou Workers' Hospital
  • Maoming People's Hospital
  • Peking University People's Hospital
  • Red Cross Hospital of Yulin City
  • Ruijin Hospital
  • The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
  • The First People's Hospital of Yunnan
  • Wuzhou People's Hospital
  • Xinqiao Hospital of Chongqing

Registry information

Important dates

Study start
2019
Primary completion
2023
Study completion
2023
First posted
Jul 5, 2019
Registry last updated
Aug 9, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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