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

Not Applicable

• Conditions: Thalassemia Major
• Interventions: Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation

• Registration Number: NCT02126046
• Lead Sponsor: Nanfang Hospital, Southern Medical University

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2012-09
• Study First Submitted: 2014-04-18
• Study First Submitted QC: 2014-04-27
• Study First Posted: 2014-04-29
• Status Verified: 2015-04
• Last Update Submitted: 2015-04-15
• Last Update Posted: 2015-04-16
• Primary Completion: 2015-12
• Study Completion: 2016-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hi-HSC-CBT	unrelated CB following haplo-identical hematopoietic stem cells transplantation	-

Primary Outcome Measures

Name	Time	Method
TM-free survival（TFS）	12 months	the measure is a composite
Transplant Related Martality (TRM)	12 months	the measure is a composite
overall survival（OS）	12 months	the measure is a composite
Primary or Secondary Graft Rejection (GR)	12 months	the measure is a composite

Secondary Outcome Measures

Name	Time	Method
The cumulative incidences of acute graft-versus-host disease（GVHD）	12 months	the measure is a composite
The cumulative incidences of chronic graft vesus host disease (cGVHD)	12 months	the measure is a composite

Trial Locations

Locations (1)

Department of paediatrics，Nangfang Hospital, Southern Medical University; 🇨🇳 Guangzhou, Guangdong, China