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

NCT04009525 | Completed Phase 4 DMC

• 1 other identifier: GX-HSCT-MT 2019
• Study Type: interventional
• Target: 823
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Thalassemia Major

Keywords

thalassemia major; hematopoietic stem cell transplantation

Outcome Measures

Primary Outcomes (2)

• Overall survival

  2-years overall survival

  2 years

• Thalassemia-free survival

  2-years thalassemia-free survival

  2 years

Secondary Outcomes (5)

• Engraftment

  30 days

• Transplant Related Mortality

  2 year

• Cumulative Incidence of acute Graft Versus Host Disease

  100 days

• Cumulative Incidence of chronic Graft Versus Host Disease

  2 years

• Cumulative Incidence of Infectious Complications

  2 years

Study Arms (3)

MSD-HSCT

EXPERIMENTAL

matched sibling donors hematopoietic stem cell transplantation

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

URD-HSCT

EXPERIMENTAL

unrelated donor hematopoietic stem cell transplantation

Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Thymoglobulin; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methotrexate

haplo-HSCT

EXPERIMENTAL

haplo-identical hematopoietic stem cell transplantation

Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Thymoglobulin; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methotrexate

Interventions

Busulfan DRUG

Busulfan（4 mg/kg/day,4 days）

Also known as: Bu

MSD-HSCT; URD-HSCT; haplo-HSCT

Cyclophosphamide DRUG

Cyclophosphamide（50 mg/kg/day,4 days）

Also known as: Cy

MSD-HSCT; URD-HSCT; haplo-HSCT

Fludarabine DRUG

Fludarabine（50 mg/m2/day,3 days）

Also known as: Flu

MSD-HSCT; URD-HSCT; haplo-HSCT

Thymoglobulin DRUG

Thymoglobulin（2.5 mg/kg/day,4 days）

Also known as: ATG

MSD-HSCT; URD-HSCT; haplo-HSCT

cyclosporine A DRUG

cyclosporine A

Also known as: CsA

MSD-HSCT

Mycophenolate mofetil DRUG

Mycophenolate mofetil（0.25g/day）

Also known as: MMF

MSD-HSCT; URD-HSCT; haplo-HSCT

Tacrolimus DRUG

Tacrolimus

Also known as: FK506

URD-HSCT; haplo-HSCT

Methotrexate DRUG

Methotrexate

Also known as: MTX

MSD-HSCT; URD-HSCT; haplo-HSCT

Eligibility Criteria

• Age: 2 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

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

Study Sites (1)

First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, 510515, China

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Busulfan; Cyclophosphamide; fludarabine; Influenza Vaccines; thymoglobulin; Cyclosporine; Mycophenolic Acid; Tacrolimus; Methotrexate

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Viral Vaccines; Vaccines; Biological Products; Complex Mixtures; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Macrolides; Lactones; Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Yongrong Lai, MD

  First Affiliated Hospital of Guangxi Medical University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of the Hematology Department

Study Record Dates

• First Submitted: July 3, 2019
• First Posted: July 5, 2019
• Study Start: July 5, 2019
• Primary Completion: July 31, 2023
• Study Completion: October 31, 2023
• Last Updated: August 9, 2024

Record last verified: 2024-08

Locations

CN (1)