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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2015 by Nanfang Hospital of Southern Medical University
Sponsor:
Information provided by (Responsible Party):
Chunfu Li, Nanfang Hospital of Southern Medical University
ClinicalTrials.gov Identifier:
NCT02126046
First received: April 18, 2014
Last updated: April 15, 2015
Last verified: April 2015
  Purpose
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 Intervention
Thalassemia Major
Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Nanfang Hospital of Southern Medical University:

Primary Outcome Measures:
  • overall survival(OS) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    the measure is a composite

  • TM-free survival(TFS) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    the measure is a composite

  • Transplant Related Martality (TRM) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    the measure is a composite

  • Primary or Secondary Graft Rejection (GR) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    the measure is a composite


Secondary Outcome Measures:
  • The cumulative incidences of acute graft-versus-host disease(GVHD) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    the measure is a composite

  • The cumulative incidences of chronic graft vesus host disease (cGVHD) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    the measure is a composite


Estimated Enrollment: 30
Study Start Date: September 2012
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Hi-HSC-CBT Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation

  Eligibility

Ages Eligible for Study:   12 Months to 18 Years   (Child, Adult)
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02126046

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

Locations
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

Additional Information:
Responsible Party: Chunfu Li, Director and professor, Nanfang Hospital of Southern Medical University
ClinicalTrials.gov Identifier: NCT02126046     History of Changes
Other Study ID Numbers: NFTM20130101 
Study First Received: April 18, 2014
Last Updated: April 15, 2015
Health Authority: China: Ethics Committee

Keywords provided by Nanfang Hospital of Southern Medical University:
Cord Blood Transplant
Haploidentical HSCT
Thalassemia major

Additional relevant MeSH terms:
Thalassemia
Beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Polystyrene sulfonic acid
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on August 25, 2016