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Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major

Primary Purpose

Thalassemia Major

Status
Unknown status
Phase
Not Applicable
Locations
China
Study Type
Interventional
Intervention
unrelated CB following haplo-identical hematopoietic stem cells transplantation
Sponsored by
Nanfang Hospital, Southern Medical University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thalassemia Major focused on measuring Cord Blood Transplant, Haploidentical HSCT, Thalassemia major

Eligibility Criteria

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

Inclusion Criteria:

  • β-thalassemia major
  • < 18 year old
  • Unrelated umbilical cord blood following Haplo-identical HSCT

Exclusion Criteria:

  • ≥ 18 year old
  • HLA- matched related donors
  • Unrelated donor transplants
  • Unrelated umbilical cord blood transplants
  • Severe iron overload in heart by T2*

Sites / Locations

  • Department of paediatrics，Nangfang Hospital, Southern Medical UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Hi-HSC-CBT

Arm Description

Outcomes

Primary Outcome Measures

overall survival（OS）
the measure is a composite
TM-free survival（TFS）
the measure is a composite
Transplant Related Martality (TRM)
the measure is a composite
Primary or Secondary Graft Rejection (GR)
the measure is a composite

Secondary Outcome Measures

The cumulative incidences of acute graft-versus-host disease（GVHD）
the measure is a composite
The cumulative incidences of chronic graft vesus host disease (cGVHD)
the measure is a composite

Full Information

First Posted
April 18, 2014
Last Updated
April 15, 2015
Sponsor
Nanfang Hospital, Southern Medical University
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1. Study Identification

Unique Protocol Identification Number
NCT02126046
Brief Title
Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
Study Type
Interventional

2. Study Status

Record Verification Date
April 2015
Overall Recruitment Status
Unknown status
Study Start Date
September 2012 (undefined)
Primary Completion Date
December 2015 (Anticipated)
Study Completion Date
March 2016 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Nanfang Hospital, Southern Medical University

4. Oversight

5. Study Description

Brief Summary
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform. But in prior Hi-HSCT using high dose post-transplant Cyclophosphamide in patients with leukemia, cytopenia post-transplant often developed, which was considered as a symptom of GVHD. Therefore, the investigators add unrelated umbilical cord blood (UCB) to the Hi-HSCT. It has reported that, as third-party cells, UCB will reduce GVHD.The purpose of this study is to determine whether unrelated UCB following Hi-HSCT can improve outcomes of Hi-HSCT in patients with β-thalassemia major.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia Major
Keywords
Cord Blood Transplant, Haploidentical HSCT, Thalassemia major

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Hi-HSC-CBT
Arm Type
Other
Intervention Type
Genetic
Intervention Name(s)
unrelated CB following haplo-identical hematopoietic stem cells transplantation
Primary Outcome Measure Information:
Title
overall survival（OS）
Description
the measure is a composite
Time Frame
12 months
Title
TM-free survival（TFS）
Description
the measure is a composite
Time Frame
12 months
Title
Transplant Related Martality (TRM)
Description
the measure is a composite
Time Frame
12 months
Title
Primary or Secondary Graft Rejection (GR)
Description
the measure is a composite
Time Frame
12 months
Secondary Outcome Measure Information:
Title
The cumulative incidences of acute graft-versus-host disease（GVHD）
Description
the measure is a composite
Time Frame
12 months
Title
The cumulative incidences of chronic graft vesus host disease (cGVHD)
Description
the measure is a composite
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Months
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: β-thalassemia major < 18 year old Unrelated umbilical cord blood following Haplo-identical HSCT Exclusion Criteria: ≥ 18 year old HLA- matched related donors Unrelated donor transplants Unrelated umbilical cord blood transplants Severe iron overload in heart by T2*
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Chunfu Li, professor
Phone
+86 20 61641921
Email
chunfugzcn@126.com
First Name & Middle Initial & Last Name or Official Title & Degree
Zhiyong Peng, Doctor
Phone
+86 20 61641925
Email
pengzhiyong8@163.com
Facility Information:
Facility Name
Department of paediatrics，Nangfang Hospital, Southern Medical University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510515
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Chunfu Li, professor
Phone
+86 20-61641921
Email
chunfugzcn@126.com

12. IPD Sharing Statement

Learn more about this trial

Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major

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