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

Thalassemia Major Clinical Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02126046
• Other study ID #: NFTM20130101
• Status: Recruiting
• Phase: N/A
• First received: April 18, 2014
• Last updated: April 15, 2015
• Start date: September 2012
• Est. completion date: March 2016

Study information

• Verified date: April 2015
• Source: Nanfang Hospital of Southern Medical University
• Contact: Chunfu Li, professor
• Phone: +86 20 61641921
• Email: chunfugzcn[@]126.com
• Is FDA regulated: No
• Health authority: China: Ethics Committee
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: March 2016
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Months to 18 Years

Eligibility

Inclusion Criteria:

• ß-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

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

Related Conditions & MeSH terms

• Beta-Thalassemia
• Thalassemia
• Thalassemia Major

Intervention

Genetic:
• unrelated CB following haplo-identical hematopoietic stem cells transplantation

Locations

Country	Name	City	State
China	Department of paediatrics,Nangfang Hospital, Southern Medical University	Guangzhou	Guangdong

Sponsors (1)

Lead Sponsor	Collaborator
Nanfang Hospital of Southern Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	overall survival(OS)	the measure is a composite	12 months	Yes
Primary	TM-free survival(TFS)	the measure is a composite	12 months	Yes
Primary	Transplant Related Martality (TRM)	the measure is a composite	12 months	Yes
Primary	Primary or Secondary Graft Rejection (GR)	the measure is a composite	12 months	Yes
Secondary	The cumulative incidences of acute graft-versus-host disease(GVHD)	the measure is a composite	12 months	Yes
Secondary	The cumulative incidences of chronic graft vesus host disease (cGVHD)	the measure is a composite	12 months	Yes

External Links

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