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Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Primary Purpose

Thalassemia Major

Status
Unknown status
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Busulfan
Cyclophosphamide
Fludarabine
Mycophenolate mofetil
Tacrolimus
Methotrexate
Thymoglobulin
Basiliximab
Sponsored by
First Affiliated Hospital of Guangxi Medical University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thalassemia Major

Eligibility Criteria

2 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Diagnosed with thalassemia major
  2. Lacking of a HLA-identical sibling donor or unrelated donor
  3. Indication of haploidentical hematopoietic stem cell transplantation
  4. No restrictions for transplantation

Exclusion Criteria:

  1. Any restriction for transplantation
  2. No indication of haploidentical hematopoietic stem cell transplantation

Sites / Locations

  • First Affiliated Hospital of Guangxi Medical UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Haploidentical HSCT

Arm Description

Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother). Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days） GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）

Outcomes

Primary Outcome Measures

Overall Survival
2-years overall survival

Secondary Outcome Measures

Disease Free Survival
2-years disease free survival
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

First Posted
April 18, 2017
Last Updated
May 27, 2017
Sponsor
First Affiliated Hospital of Guangxi Medical University
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1. Study Identification

Unique Protocol Identification Number
NCT03171831
Brief Title
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
Official Title
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
Study Type
Interventional

2. Study Status

Record Verification Date
May 2017
Overall Recruitment Status
Unknown status
Study Start Date
April 1, 2017 (Actual)
Primary Completion Date
December 31, 2021 (Anticipated)
Study Completion Date
April 1, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
First Affiliated Hospital of Guangxi Medical University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
Detailed Description
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia Major

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Haploidentical HSCT
Arm Type
Experimental
Arm Description
Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother). Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days） GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4）
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)
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（0.03mg/kg/day）
Intervention Type
Drug
Intervention Name(s)
Methotrexate
Other Intervention Name(s)
MTX
Intervention Description
Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）
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)
Basiliximab
Other Intervention Name(s)
Simulect
Intervention Description
Basiliximab（10mg on day 0 and +4）
Primary Outcome Measure Information:
Title
Overall Survival
Description
2-years overall survival
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Disease Free Survival
Description
2-years disease free survival
Time Frame
2 years
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
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Diagnosed with thalassemia major Lacking of a HLA-identical sibling donor or unrelated donor Indication of haploidentical hematopoietic stem cell transplantation No restrictions for transplantation Exclusion Criteria: Any restriction for transplantation No indication of haploidentical hematopoietic stem cell 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
Meiqing Wu, MD
Phone
+86（0771）5356510
Email
wumeiqing1309@163.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

12. IPD Sharing Statement

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Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

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