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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02126046
Recruitment Status : Unknown
Verified April 2015 by Chunfu Li, Nanfang Hospital of Southern Medical University.
Recruitment status was:  Recruiting
First Posted : April 29, 2014
Last Update Posted : April 16, 2015
Sponsor:
Information provided by (Responsible Party):
Chunfu Li, Nanfang Hospital of Southern Medical University

Study Description
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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.

Condition or disease Intervention/treatment Phase
Thalassemia Major Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : September 2012
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : March 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Hi-HSC-CBT Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation


Outcome Measures
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Primary Outcome Measures :
  1. overall survival（OS） [ Time Frame: 12 months ]
    the measure is a composite

  2. TM-free survival（TFS） [ Time Frame: 12 months ]
    the measure is a composite

  3. Transplant Related Martality (TRM) [ Time Frame: 12 months ]
    the measure is a composite

  4. Primary or Secondary Graft Rejection (GR) [ Time Frame: 12 months ]
    the measure is a composite


Secondary Outcome Measures :
  1. The cumulative incidences of acute graft-versus-host disease（GVHD） [ Time Frame: 12 months ]
    the measure is a composite

  2. The cumulative incidences of chronic graft vesus host disease (cGVHD) [ Time Frame: 12 months ]
    the measure is a composite


Eligibility Criteria
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Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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*
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02126046


Contacts
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Contact: Chunfu Li, professor +86 20 61641921 chunfugzcn@126.com
Contact: Zhiyong Peng, Doctor +86 20 61641925 pengzhiyong8@163.com

Locations
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China, Guangdong
Department of paediatrics，Nangfang Hospital, Southern Medical University Recruiting
Guangzhou, Guangdong, China, 510515
Contact: Chunfu Li, professor    +86 20-61641921    chunfugzcn@126.com   
Sponsors and Collaborators
Nanfang Hospital of Southern Medical University
More Information
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Responsible Party: Chunfu Li, Director and professor, Nanfang Hospital of Southern Medical University
ClinicalTrials.gov Identifier: NCT02126046    
Other Study ID Numbers: NFTM20130101
First Posted: April 29, 2014    Key Record Dates
Last Update Posted: April 16, 2015
Last Verified: April 2015
Keywords provided by Chunfu Li, Nanfang Hospital of Southern Medical University:
Cord Blood Transplant
Haploidentical HSCT
Thalassemia major
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn