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

April 15, 2015 updated by: Chunfu Li, Nanfang Hospital of Southern Medical University
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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Recruiting
        • Department of paediatrics，Nangfang Hospital, Southern Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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*

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Hi-HSC-CBT
Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall survival（OS）
Time Frame: 12 months
the measure is a composite
12 months
TM-free survival（TFS）
Time Frame: 12 months
the measure is a composite
12 months
Transplant Related Martality (TRM)
Time Frame: 12 months
the measure is a composite
12 months
Primary or Secondary Graft Rejection (GR)
Time Frame: 12 months
the measure is a composite
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The cumulative incidences of acute graft-versus-host disease（GVHD）
Time Frame: 12 months
the measure is a composite
12 months
The cumulative incidences of chronic graft vesus host disease (cGVHD)
Time Frame: 12 months
the measure is a composite
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nanfang Hospital of Southern Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2012

Primary Completion (Anticipated)

December 1, 2015

Study Completion (Anticipated)

March 1, 2016

Study Registration Dates

First Submitted

April 18, 2014

First Submitted That Met QC Criteria

April 27, 2014

First Posted (Estimate)

April 29, 2014

Study Record Updates

Last Update Posted (Estimate)

April 16, 2015

Last Update Submitted That Met QC Criteria

April 15, 2015

Last Verified

April 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NFTM20130101

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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