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出境医 / 临床实验 / Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

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

Study Description
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.
Study Design
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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
Arms and Interventions
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
Outcome Measures
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


Eligibility Criteria
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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.
Contacts and Locations

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
Layout table for investigator information
Principal Investigator: Yongrong Lai, MD First Affiliated Hospital of Guangxi Medical University
Tracking Information
First Submitted Date  ICMJE July 3, 2019
First Posted Date  ICMJE July 5, 2019
Last Update Posted Date July 5, 2019
Actual Study Start Date  ICMJE June 1, 2019
Estimated Primary Completion Date May 31, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 3, 2019)
  • Overall survival [ Time Frame: 2 years ]
    2-years overall survival
  • Thalassemia-free survival [ Time Frame: 2 years ]
    2-years thalassemia-free survival
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: July 3, 2019)
  • Engraftment [ Time Frame: 30 days ]
    Myeloid engraftment at day +30
  • Transplant Related Mortality [ Time Frame: 1 year ]
    Transplant-related mortality by 1 year
  • Cumulative Incidence of acute Graft Versus Host Disease [ Time Frame: 180 days ]
    Acute graft versus host disease at day +180
  • Cumulative Incidence of chronic Graft Versus Host Disease [ Time Frame: 2 years ]
    Chronic graft versus host disease by 2 years
  • Cumulative Incidence of Infectious Complications [ Time Frame: 2 years ]
    Cumulative incidence of bacterial, fungal and viral infections by 2 years
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Official Title  ICMJE Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Thalassemia Major
Intervention  ICMJE
  • 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: Tacrolimus
    Tacrolimus
    Other Name: FK506
  • Drug: Methotrexate
    Methotrexate
    Other Name: MTX
  • Drug: Basiliximab
    Basiliximab
    Other Name: Simulect
  • Drug: Ruxolitinib
    Ruxolitinib
Study Arms  ICMJE
  • Experimental: MSD-HSCT
    matched sibling donors hematopoietic stem cell transplantation
    Interventions:
    • Drug: Busulfan
    • Drug: Cyclophosphamide
    • Drug: Fludarabine
    • Drug: Thymoglobulin
    • Drug: cyclosporine A
    • Drug: Mycophenolate mofetil
    • Drug: Methotrexate
  • Experimental: URD-HSCT
    unrelated donor hematopoietic stem cell transplantation
    Interventions:
    • Drug: Busulfan
    • Drug: Cyclophosphamide
    • Drug: Fludarabine
    • Drug: Thymoglobulin
    • Drug: Mycophenolate mofetil
    • Drug: Tacrolimus
    • Drug: Methotrexate
    • Drug: Ruxolitinib
  • Experimental: haplo-HSCT
    haplo-identical hematopoietic stem cell transplantation
    Interventions:
    • Drug: Busulfan
    • Drug: Cyclophosphamide
    • Drug: Fludarabine
    • Drug: Thymoglobulin
    • Drug: Mycophenolate mofetil
    • Drug: Tacrolimus
    • Drug: Methotrexate
    • Drug: Basiliximab
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 3, 2019) 		800
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE May 31, 2023
Estimated Primary Completion Date May 31, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 20 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Yongrong Lai, MD +86（0771）5356510 laiyongrong@hotmail.com
Contact: Qiaochuan Li, MD +86（0771）5356510 2402576451@qq.com
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04009525
Other Study ID Numbers  ICMJE GX-HSCT-MT 2019
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Yongrong Lai, First Affiliated Hospital of Guangxi Medical University
Study Sponsor  ICMJE First Affiliated Hospital of Guangxi Medical University
Collaborators  ICMJE
  • 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  ICMJE
Principal Investigator: Yongrong Lai, MD First Affiliated Hospital of Guangxi Medical University
PRS Account First Affiliated Hospital of Guangxi Medical University
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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