Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
1 other identifier
interventional
30
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Sep 2012
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2012
CompletedFirst Submitted
Initial submission to the registry
April 18, 2014
CompletedFirst Posted
Study publicly available on registry
April 29, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2016
CompletedApril 16, 2015
April 1, 2015
3.2 years
April 18, 2014
April 15, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (4)
overall survival（OS）
the measure is a composite
12 months
TM-free survival（TFS）
the measure is a composite
12 months
Transplant Related Martality (TRM)
the measure is a composite
12 months
Primary or Secondary Graft Rejection (GR)
the measure is a composite
12 months
Secondary Outcomes (2)
The cumulative incidences of acute graft-versus-host disease（GVHD）
12 months
The cumulative incidences of chronic graft vesus host disease (cGVHD)
12 months
Study Arms (1)
Hi-HSC-CBT
OTHERInterventions
Eligibility Criteria
You may qualify if:
- β-thalassemia major
- \< 18 year old
- Unrelated umbilical cord blood following Haplo-identical HSCT
You may not qualify if:
- ≥ 18 year old
- HLA- matched related donors
- Unrelated donor transplants
- Unrelated umbilical cord blood transplants
- Severe iron overload in heart by T2\*
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Department of paediatrics，Nangfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Director and professor
Study Record Dates
First Submitted
April 18, 2014
First Posted
April 29, 2014
Study Start
September 1, 2012
Primary Completion
December 1, 2015
Study Completion
March 1, 2016
Last Updated
April 16, 2015
Record last verified: 2015-04