NCT00029380

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

Sibling Donor Cord Blood Banking and Transplantation

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: National Heart, Lung, and Blood Institute (NHLBI) · NIH
• Sex: All
• Age: 3 Years – 14 Years
• Healthy volunteers: Not accepted

Summary

This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia.

Detailed description

BACKGROUND: During the past decade, a number of advances have been made in the treatment of patients with sickle cell anemia and thalassemia. Among these advances is allogeneic bone marrow transplantation, which is the only current treatment that offers a potential for cure. In sickle cell anemia, transplantation has been performed in patients who have had advanced organ damage. In thalassemia, transplantation has been performed before having any evidence of iron-related tissue damage. Due to concerns over engraftment and graft versus host disease (GVHD), transplants for patients with hemoglobinopathies have been limited to situations in which a human leukocyte antigen (HLA) compatible donor existed. Unfortunately, an HLA-matched related donor is often not available. Umbilical cord blood (UCB), a recently recognized source of hematopoietic stem cells, has been used to successfully transplant bone marrow to over 500 patients. The potential advantage of cord blood over other donor sources of stem cells is the minimal risk of high-grade GVHD (even without complete HLA compatibility). DESIGN NARRATIVE: This study will establish a national sibling donor cord blood (SDCB) program, evaluate its use in a multi-center pilot study of transplantation, and develop a Web-based data management system to support these two projects. A multi-center pilot study was conducted on cord blood transplantation in children with either sickle cell disease or thalassemia. The investigators tested the hypothesis that a novel immunosuppressive conditioning regimen (fludarabine, cyclophosphamide, and busulfan) and post transplant therapy (mycophenolate mofetil and cyclosporine) would improve engraftment rates and prevent disease recurrence. The effect of SDCB transplantation on hematologic parameters and GVHD was monitored. Enrollment in the study was suspended on December 29, 2003. The protocol was revised, replacing the previous conditioning regimen of fludarabine, busulfan, and cyclophosphamide with a more conventional regimen of rabbit anti-thymocyte globulin (Sangstat), busulfan, and cyclophosphamide. The revised protocol is open for enrollment.

Conditions

• Hematologic Diseases
• Anemia, Sickle Cell
• Beta-Thalassemia
• Hematopoietic Stem Cell Transplantation

Interventions

Type	Name	Description
DRUG	Sangstat
DRUG	Cyclophosphamide
DRUG	Busulfan
DRUG	Mycophenolate Mofetil
DRUG	Cyclosporine
PROCEDURE	Cord Blood Transplantation

Timeline

• Start date: 1999-01-01
• Primary completion: 2006-08-01
• Completion: 2006-08-01
• First posted: 2002-01-11
• Last updated: 2016-07-29

Locations

15 sites across 2 countries: United States, Canada