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Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

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. Identifier: NCT00029380
Recruitment Status : Completed
First Posted : January 11, 2002
Last Update Posted : July 29, 2016
Information provided by:
National Heart, Lung, and Blood Institute (NHLBI)

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

Condition or disease Intervention/treatment Phase
Hematologic Diseases Anemia, Sickle Cell Beta-Thalassemia Hematopoietic Stem Cell Transplantation Drug: Sangstat Drug: Cyclophosphamide Drug: Busulfan Drug: Mycophenolate Mofetil Drug: Cyclosporine Procedure: Cord Blood Transplantation Phase 2

Detailed Description:


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).


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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Primary Purpose: Treatment
Official Title: Sibling Donor Cord Blood Banking and Transplantation
Study Start Date : January 1999
Actual Primary Completion Date : August 2006
Actual Study Completion Date : August 2006

Primary Outcome Measures :
  1. Hematologic parameters
  2. GVHD

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   3 Years to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Suitable UCB collection from an HLA-identical sibling
  • Sickle cell anemia (Hb SS or S beta thalassemia) with significant disease manifestations as defined by at least one of the following criteria:

    1. A history of painful events defined as three or more painful events in the 2 years prior to enrollment. Pain may occur in typical sites associated with vaso-occlusive painful events and cannot be explained by causes other than sickle cell disease. The pain must last at least 4 hours and require treatment with either parenteral narcotics, an equianalgesic dose of oral narcotics (if pain is treated in a local facility where parenteral narcotics are not routinely used to treat painful events), or parenteral nonsteroidal anti-inflammatory drugs. Painful events managed at home will be considered only if there is documentation of the event in a clinical record that may be reviewed by an investigator.
    2. Acute chest syndrome (ACS) with two or more episodes of ACS with the development of a new infiltrate on chest radiograph and/or having a perfusion defect demonstrable on a lung radioisotope scan
    3. Any combination of painful events and episodes of ACS that total three events in the 2 years before transplantation
    4. Any clinically significant neurologic event (stroke or hemorrhage) or any neurologic defect lasting more than 24 hours
    5. Abnormal cerebral MRI and abnormal cerebral MRA
    6. An episode of dactylitis in the first year of life with significant anemia (Hbg less than 7 g/dL), or leukocytosis in the second year of life such that the risk of a severe adverse outcome before 18 years of age exceeds 54% (as defined by the cooperative study of sickle cell disease (CSSCD) infant cohort study)
    7. History of positive trans-cranial Doppler studies (average greater than 200 cm/sec)
  • Beta thalassemia major with significant disease manifestations as defined by the following criteria: Beta thalassemia genotype consistent with clinical diagnosis of beta thalassemia major (could include patients with E-beta thalassemia genotype) and requiring eight or more red blood cell (RBC) transfusions a year and iron chelation therapy. Younger patients who are at risk of transfusional iron overload but who have not yet initiated iron chelation therapy will be eligible.
  • Adequate physical function as measured by the following criteria:

    1. Cardiac: Asymptomatic or, if symptomatic, then left ventricular ejection fraction at rest must be greater than 40% and must improve with exercise, or shortening fraction greater than 26%
    2. Hepatic: Less than 5 times the clinical baseline of AST and less than 2.5 times the clinical baseline mg/dL of total serum bilirubin (clinical baseline is determined from the mean of the four most recent test results)
    3. Renal: Serum creatinine within normal range for age or if serum creatinine is outside normal range for age then renal function (creatinine clearance or GFR) greater than 50% of the lower limit of normal (LLN) for age
    4. Pulmonary: Asymptomatic, or, if symptomatic, DLCO, FEV1, FEC (diffusion capacity) greater than 45% of predicted (corrected for hemoglobin); if unable to obtain PFT, oxygen saturation greater than 85% on room air

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 identifier (NCT number): NCT00029380

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United States, California
Children's Hospital Oakland
Oakland, California, United States, 94609
Children's Hospital, Oakland
Oakland, California, United States, 94609
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States
United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
University of Miami Batchelor Children's Research Center
Miami, Florida, United States, 33136
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, Louisiana
Louisiana State University Children's Medical Center
New Orleans, Louisiana, United States
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, New Jersey
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, North Carolina
Duke University Medical Center Children's Hospital
Durham, North Carolina, United States
United States, Pennsylvania
Children's Hospital Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29403
United States, Texas
University of Texas Southwestern Medical Center - Dallas
Dallas, Texas, United States, 75235
Texas Transplant Institute
San Antonio, Texas, United States, 78229
Canada, Quebec
Hopital Ste-Justine
Montreal, Quebec, Canada
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
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Study Chair: Victor Aquino University of Texas Southwestern Medical Center - Dallas
Study Chair: Nancy Bunin Children's Hospital of Philadelphia
Study Chair: Martin Champagne Hopital Ste-Justine
Study Chair: Joel Brochstein Hackensack Meridian Health
Study Chair: Michael Joyce Nemours Children's Clinic
Study Chair: Naynesh Kamani Children's National Research Institute
Study Chair: Gary Kleiner University of Miami Batchelor Children's Research Center
Study Chair: Joanne Kurtzberg Duke University Medical Center Children's Hospital
Study Chair: Bertram H. Lubin UCSF Benioff Children's Hospital Oakland
Study Chair: Alexis Thompson Ann & Robert H Lurie Children's Hospital of Chicago
Study Chair: Donna Wall Texas Transplant Institute
Study Chair: Mark Walters UCSF Benioff Children's Hospital Oakland
Study Chair: Lolie Yu Louisiana State University Children's Medical Center

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Responsible Party: Bertram H. Lubin, Children's Hospital, Oakland Identifier: NCT00029380    
Other Study ID Numbers: 141
U01HL061877 ( U.S. NIH Grant/Contract )
First Posted: January 11, 2002    Key Record Dates
Last Update Posted: July 29, 2016
Last Verified: September 2008
Additional relevant MeSH terms:
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Hematologic Diseases
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Mycophenolic Acid
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Enzyme Inhibitors
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Calcineurin Inhibitors
Antibiotics, Antineoplastic
Antibiotics, Antitubercular