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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04009525
Recruitment Status : Recruiting
First Posted : July 5, 2019
Last Update Posted : July 5, 2019
Sponsor:
Collaborators:
Peking University People's Hospital
Ruijin Hospital
The 923th Hospital of People's Liberation Army
Fourth Affiliated Hospital of Guangxi Medical University
Liuzhou General Hospital
Hainan General Hospital
The Affiliated Hospital Of Guizhou Medical University
The First People's Hospital of Yunnan
Information provided by (Responsible Party):
Yongrong Lai, First Affiliated Hospital of Guangxi Medical University

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.

Condition or disease Intervention/treatment Phase
Thalassemia Major Drug: Busulfan Drug: Cyclophosphamide Drug: Fludarabine Drug: Thymoglobulin Drug: cyclosporine A Drug: Mycophenolate mofetil Drug: Tacrolimus Drug: Methotrexate Drug: Basiliximab Drug: Ruxolitinib Phase 4

Detailed 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 800 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Actual Study Start Date : June 1, 2019
Estimated Primary Completion Date : May 31, 2022
Estimated Study Completion Date : May 31, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arm Intervention/treatment
Experimental: MSD-HSCT
matched sibling donors hematopoietic stem cell transplantation
Drug: Busulfan
Busulfan(4 mg/kg/day,4 days)
Other Name: Bu

Drug: Cyclophosphamide
Cyclophosphamide(50 mg/kg/day,4 days)
Other Name: Cy

Drug: Fludarabine
Fludarabine(50 mg/m2/day,3 days)
Other Name: Flu

Drug: Thymoglobulin
Thymoglobulin(2.5 mg/kg/day,4 days)
Other Name: ATG

Drug: cyclosporine A
cyclosporine A
Other Name: CsA

Drug: Mycophenolate mofetil
Mycophenolate mofetil(0.25g/day)
Other Name: MMF

Drug: Methotrexate
Methotrexate
Other Name: MTX

Experimental: URD-HSCT
unrelated donor hematopoietic stem cell transplantation
Drug: Busulfan
Busulfan(4 mg/kg/day,4 days)
Other Name: Bu

Drug: Cyclophosphamide
Cyclophosphamide(50 mg/kg/day,4 days)
Other Name: Cy

Drug: Fludarabine
Fludarabine(50 mg/m2/day,3 days)
Other Name: Flu

Drug: Thymoglobulin
Thymoglobulin(2.5 mg/kg/day,4 days)
Other Name: ATG

Drug: Mycophenolate mofetil
Mycophenolate mofetil(0.25g/day)
Other Name: MMF

Drug: Tacrolimus
Tacrolimus
Other Name: FK506

Drug: Methotrexate
Methotrexate
Other Name: MTX

Drug: Ruxolitinib
Ruxolitinib

Experimental: haplo-HSCT
haplo-identical hematopoietic stem cell transplantation
Drug: Busulfan
Busulfan(4 mg/kg/day,4 days)
Other Name: Bu

Drug: Cyclophosphamide
Cyclophosphamide(50 mg/kg/day,4 days)
Other Name: Cy

Drug: Fludarabine
Fludarabine(50 mg/m2/day,3 days)
Other Name: Flu

Drug: Thymoglobulin
Thymoglobulin(2.5 mg/kg/day,4 days)
Other Name: ATG

Drug: Mycophenolate mofetil
Mycophenolate mofetil(0.25g/day)
Other Name: MMF

Drug: Tacrolimus
Tacrolimus
Other Name: FK506

Drug: Methotrexate
Methotrexate
Other Name: MTX

Drug: Basiliximab
Basiliximab
Other Name: Simulect




Primary Outcome Measures :
  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 Outcome Measures :
  1. Engraftment [ Time Frame: 30 days ]
    Myeloid engraftment at day +30

  2. Transplant Related Mortality [ Time Frame: 1 year ]
    Transplant-related mortality by 1 year

  3. Cumulative Incidence of acute Graft Versus Host Disease [ Time Frame: 180 days ]
    Acute graft versus host disease at day +180

  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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   2 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04009525


Contacts
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Contact: Yongrong Lai, MD +86(0771)5356510 laiyongrong@hotmail.com
Contact: Qiaochuan Li, MD +86(0771)5356510 2402576451@qq.com

Locations
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China, Guangxi
First Affiliated Hospital of Guangxi Medical University Recruiting
Nanning, Guangxi, China, 510515
Contact: Yongrong Lai, MD    +86(0771)5356510    laiyongrong@hotmail.com   
Contact: Meiqing Wu, MD    +86(0771)5356510    453433378@qq.com   
Sub-Investigator: Jianming Luo, MD         
Sub-Investigator: Xiaojun Huang, MD         
Sub-Investigator: Jiong Hu, MD         
Sub-Investigator: Xiaolin Yin, MD         
Sub-Investigator: Hong Chen, MD         
Sub-Investigator: Jinxiong Huang, MD         
Sub-Investigator: Hongxia Yao, MD         
Sub-Investigator: Jishi Wang, MD         
Sub-Investigator: Tonghua Yang, MD         
Sponsors and Collaborators
First Affiliated Hospital of Guangxi Medical University
Peking University People's Hospital
Ruijin Hospital
The 923th Hospital of People's Liberation Army
Fourth Affiliated Hospital of Guangxi Medical University
Liuzhou General Hospital
Hainan General Hospital
The Affiliated Hospital Of Guizhou Medical University
The First People's Hospital of Yunnan
Investigators
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Principal Investigator: Yongrong Lai, MD First Affiliated Hospital of Guangxi Medical University

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Responsible Party: Yongrong Lai, Director of the Hematology Department, First Affiliated Hospital of Guangxi Medical University
ClinicalTrials.gov Identifier: NCT04009525    
Other Study ID Numbers: GX-HSCT-MT 2019
First Posted: July 5, 2019    Key Record Dates
Last Update Posted: July 5, 2019
Last Verified: July 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Yongrong Lai, First Affiliated Hospital of Guangxi Medical University:
thalassemia major
hematopoietic stem cell transplantation
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Cyclosporine
Mycophenolic Acid
Cyclophosphamide
Busulfan
Methotrexate
Fludarabine
Fludarabine phosphate
Tacrolimus
Cyclosporins
Thymoglobulin
Basiliximab
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Abortifacient Agents, Nonsteroidal
Abortifacient Agents