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Clinical Trial: Sibling Donor Cord Blood Banking and Transplantation
This study is not yet open for patient recruitment.
Purpose
To develop a national cord blood bank for siblings of patients with hemoglobinopathies, thalassemia and potentially malignant diseases.
| Condition | Treatment or Intervention |
|---|---|
| Blood Disease Anemia, Sickle Cell Anemia (Cooley's) Leukemia Bone Marrow Transplantation Hematopoietic Stem Cell Transplantation | Drug: fludarabine Drug: cyclophosphamide Drug: busulfan Drug: mycophenolate mofetil Drug: cyclosporine Procedure: cord blood transplantation |
MedlinePlus related topics: Anemia; Blood and Blood Disorders; Leukemia, Adult Acute; Leukemia, Adult Chronic; Leukemia, Childhood; Sickle Cell Anemia
Genetics Home Reference related topics: beta thalassemia; sickle cell anemia
Study Type: Interventional
Study Design: Treatment
Study start: January 1999; Expected completion: August 2005
BACKGROUND: During the past decade, a number of advances have been made in the treatment or patients sickle cell anemia and thalassemia. Among these is allogeneic bone marrow transplantation, the only current treatment that offers the potential for cure. In sickle cell anemia, transplantation has been performed in patients who have had advanced organ damage while in thalassemia, transplantation has been performed before 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 where an HLA compatible donor exists. Unfortunately, an HLA matched related donor is often not available. Umbilical cord blood, a recently recognized source of hematopoietic stem cells, has been used to successfully transplant over 500 patients. The potential advantages of cord blood over other donor sources of stem cells is related to the observation that even without complete HLA compatibility, the risk of high-grade GVHD is minimal.
DESIGN NARRATIVE: The study established a national Sibling Donor Cord Blood (SDCB) Program, evaluated its use in a multi-center pilot study of transplantation, and developed 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 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.
Eligibility
Genders Eligible for Study: Both
Criteria
Location Information
Bertram Lubin, Children's Hospital, Oakland
More Information
Publications
Reed W, Walters M, Lubin BH. Collection of sibling donor cord blood for children with thalassemia. J Pediatr Hematol Oncol. 2000 Nov-Dec;22(6):602-4.
Lubin BH, Eraklis M, Apicelli G. Umbilical cord blood banking. Adv Pediatr. 1999;46:383-408. Review. No abstract available.
Woodard P, Lubin B, Walters CM. New approaches to hematopoietic cell transplantation for hematological diseases in children. Pediatr Clin North Am. 2002 Oct;49(5):989-1007. Review.
Reed W, Smith R, Dekovic F, Lee JY, Saba JD, Trachtenberg E, Epstein J, Haaz S, Walters MC, Lubin BH. Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease. Blood. 2003 Jan 1;101(1):351-7.
Locatelli F, Rocha V, Reed W, Bernaudin F, Ertem M, Grafakos S, Brichard B, Li X, Nagler A, Giorgiani G, Haut PR, Brochstein JA, Nugent DJ, Blatt J, Woodard P, Kurtzberg J, Rubin CM, Miniero R, Lutz P, Raja T, Roberts I, Will AM, Yaniv I, Vermylen C, Tannoia N, Garnier F, Ionescu I, Walters MC, Lubin BH, Gluckman E. Related umbilical cord blood transplant in patients with Thalassemia and Sickle Cell Disease. Blood. 2002 Nov 7 [epub ahead of print]
Reed W, Smith R, Dekovic F, Lee JY, Saba JD, Trachtenberg E, Epstein J, Haaz S, Walters MC, Lubin BH. Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease. Blood. 2003 Jan 1;101(1):351-7.
Reed W, Walters M, Trachtenberg E, Smith R, Lubin BH. Sibling donor cord blood banking for children with sickle cell disease. Pediatr Pathol Mol Med. 2001 Mar-Apr;20(2):167-74.
Locatelli F, Rocha V, Reed W, Bernaudin F, Ertem M, Grafakos S, Brichard B, Li X, Nagler A, Giorgiani G, Haut PR, Brochstein JA, Nugent DJ, Blatt J, Woodard P, Kurtzberg J, Rubin CM, Miniero R, Lutz P, Raja T, Roberts I, Will AM, Yaniv I, Vermylen C, Tannoia N, Garnier F, Ionescu I, Walters MC, Lubin BH, Gluckman E. Related umbilical cord blood transplant in patients with Thalassemia and Sickle Cell Disease. Blood. 2002 Nov 7 [epub ahead of print]
Record last reviewed: March 2005
Last Updated: March 18, 2005
Record first received: January 10, 2002
ClinicalTrials.gov Identifier: NCT00029380
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08
Source: ClinicalTrials.gov
Cache Date: April 9, 2005
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