Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Primary Purpose
Thalassemia Major
Status
Unknown status
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Busulfan
Cyclophosphamide
Fludarabine
Thymoglobulin
cyclosporine A
Mycophenolate mofetil
Tacrolimus
Methotrexate
Basiliximab
Ruxolitinib
Sponsored by
About this trial
This is an interventional treatment trial for Thalassemia Major focused on measuring thalassemia major, hematopoietic stem cell transplantation
Eligibility Criteria
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.
Sites / Locations
- First Affiliated Hospital of Guangxi Medical UniversityRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
MSD-HSCT
URD-HSCT
haplo-HSCT
Arm Description
matched sibling donors hematopoietic stem cell transplantation
unrelated donor hematopoietic stem cell transplantation
haplo-identical hematopoietic stem cell transplantation
Outcomes
Primary Outcome Measures
Overall survival
2-years overall survival
Thalassemia-free survival
2-years thalassemia-free survival
Secondary Outcome Measures
Engraftment
Myeloid engraftment at day +30
Transplant Related Mortality
Transplant-related mortality by 1 year
Cumulative Incidence of acute Graft Versus Host Disease
Acute graft versus host disease at day +180
Cumulative Incidence of chronic Graft Versus Host Disease
Chronic graft versus host disease by 2 years
Cumulative Incidence of Infectious Complications
Cumulative incidence of bacterial, fungal and viral infections by 2 years
Full Information
NCT ID
NCT04009525
First Posted
July 3, 2019
Last Updated
July 3, 2019
Sponsor
First Affiliated Hospital of Guangxi Medical University
Collaborators
Peking University People's Hospital, Ruijin Hospital, The 923rd Hospital of Joint Logistics Support Force 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
1. Study Identification
Unique Protocol Identification Number
NCT04009525
Brief Title
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Official Title
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
Study Type
Interventional
2. Study Status
Record Verification Date
July 2019
Overall Recruitment Status
Unknown status
Study Start Date
June 1, 2019 (Actual)
Primary Completion Date
May 31, 2022 (Anticipated)
Study Completion Date
May 31, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
First Affiliated Hospital of Guangxi Medical University
Collaborators
Peking University People's Hospital, Ruijin Hospital, The 923rd Hospital of Joint Logistics Support Force 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
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia Major
Keywords
thalassemia major, hematopoietic stem cell transplantation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
800 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
MSD-HSCT
Arm Type
Experimental
Arm Description
matched sibling donors hematopoietic stem cell transplantation
Arm Title
URD-HSCT
Arm Type
Experimental
Arm Description
unrelated donor hematopoietic stem cell transplantation
Arm Title
haplo-HSCT
Arm Type
Experimental
Arm Description
haplo-identical hematopoietic stem cell transplantation
Intervention Type
Drug
Intervention Name(s)
Busulfan
Other Intervention Name(s)
Bu
Intervention Description
Busulfan(4 mg/kg/day,4 days)
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Other Intervention Name(s)
Cy
Intervention Description
Cyclophosphamide(50 mg/kg/day,4 days)
Intervention Type
Drug
Intervention Name(s)
Fludarabine
Other Intervention Name(s)
Flu
Intervention Description
Fludarabine(50 mg/m2/day,3 days)
Intervention Type
Drug
Intervention Name(s)
Thymoglobulin
Other Intervention Name(s)
ATG
Intervention Description
Thymoglobulin(2.5 mg/kg/day,4 days)
Intervention Type
Drug
Intervention Name(s)
cyclosporine A
Other Intervention Name(s)
CsA
Intervention Description
cyclosporine A
Intervention Type
Drug
Intervention Name(s)
Mycophenolate mofetil
Other Intervention Name(s)
MMF
Intervention Description
Mycophenolate mofetil(0.25g/day)
Intervention Type
Drug
Intervention Name(s)
Tacrolimus
Other Intervention Name(s)
FK506
Intervention Description
Tacrolimus
Intervention Type
Drug
Intervention Name(s)
Methotrexate
Other Intervention Name(s)
MTX
Intervention Description
Methotrexate
Intervention Type
Drug
Intervention Name(s)
Basiliximab
Other Intervention Name(s)
Simulect
Intervention Description
Basiliximab
Intervention Type
Drug
Intervention Name(s)
Ruxolitinib
Intervention Description
Ruxolitinib
Primary Outcome Measure Information:
Title
Overall survival
Description
2-years overall survival
Time Frame
2 years
Title
Thalassemia-free survival
Description
2-years thalassemia-free survival
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Engraftment
Description
Myeloid engraftment at day +30
Time Frame
30 days
Title
Transplant Related Mortality
Description
Transplant-related mortality by 1 year
Time Frame
1 year
Title
Cumulative Incidence of acute Graft Versus Host Disease
Description
Acute graft versus host disease at day +180
Time Frame
180 days
Title
Cumulative Incidence of chronic Graft Versus Host Disease
Description
Chronic graft versus host disease by 2 years
Time Frame
2 years
Title
Cumulative Incidence of Infectious Complications
Description
Cumulative incidence of bacterial, fungal and viral infections by 2 years
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yongrong Lai, MD
Phone
+86(0771)5356510
Email
laiyongrong@hotmail.com
First Name & Middle Initial & Last Name or Official Title & Degree
Qiaochuan Li, MD
Phone
+86(0771)5356510
Email
2402576451@qq.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Yongrong Lai, MD
Organizational Affiliation
First Affiliated Hospital of Guangxi Medical University
Official's Role
Principal Investigator
Facility Information:
Facility Name
First Affiliated Hospital of Guangxi Medical University
City
Nanning
State/Province
Guangxi
ZIP/Postal Code
510515
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yongrong Lai, MD
Phone
+86(0771)5356510
Email
laiyongrong@hotmail.com
First Name & Middle Initial & Last Name & Degree
Meiqing Wu, MD
Phone
+86(0771)5356510
Email
453433378@qq.com
First Name & Middle Initial & Last Name & Degree
Jianming Luo, MD
First Name & Middle Initial & Last Name & Degree
Xiaojun Huang, MD
First Name & Middle Initial & Last Name & Degree
Jiong Hu, MD
First Name & Middle Initial & Last Name & Degree
Xiaolin Yin, MD
First Name & Middle Initial & Last Name & Degree
Hong Chen, MD
First Name & Middle Initial & Last Name & Degree
Jinxiong Huang, MD
First Name & Middle Initial & Last Name & Degree
Hongxia Yao, MD
First Name & Middle Initial & Last Name & Degree
Jishi Wang, MD
First Name & Middle Initial & Last Name & Degree
Tonghua Yang, MD
12. IPD Sharing Statement
Learn more about this trial
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
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