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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

About this trial

This is an interventional treatment trial for Thalassemia Major focused on measuring thalassemia major, hematopoietic stem cell transplantation

Eligibility Criteria

2 Years - 20 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

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

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

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)

Intervention Description

Busulfan（4 mg/kg/day,4 days）

Intervention Type

Drug

Intervention Name(s)

Cyclophosphamide

Other Intervention Name(s)

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

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

laiyongrong@hotmail.com

First Name & Middle Initial & Last Name or Official Title & Degree

Qiaochuan Li, MD

Phone

+86（0771）5356510

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

laiyongrong@hotmail.com

First Name & Middle Initial & Last Name & Degree

Meiqing Wu, MD

Phone

+86（0771）5356510

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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