Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

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: NCT00004485
Recruitment Status : Completed
First Posted : October 19, 1999
Last Update Posted : September 10, 2008
Information provided by:
Office of Rare Diseases (ORD)

Brief Summary:

RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease.

PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Anemia Drug: cyclosporine Drug: fludarabine Drug: mycophenolate mofetil Procedure: Bone Marrow Transplantation Phase 1 Phase 2

Detailed Description:

PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27.

Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.

Study Type : Interventional  (Clinical Trial)
Enrollment : 50 participants
Primary Purpose: Treatment
Official Title: Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease
Study Start Date : December 1999
Actual Primary Completion Date : August 2007

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Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

Diagnosis of sickle cell anemia with clinically severe disease manifestations defined by: Recurrent painful events (at least 2 painful events in past year) which cannot be explained by other causes Pain lasts at least 4 hours Requires treatment with parenteral narcotics, equianalgesic dose of oral narcotics, or parenteral nonsteroidal antiinflammatory drugs Acute chest syndrome (ACS) with at least 2 episodes within past 2 years that required hospitalization, oxygen, and RBC transfusion Any combination of painful events and ACS episodes that total 2 events within the past year Abnormal cerebral MRI, abnormal angiography (MR or conventional), and abnormal neuropsychologic testing performance

No stage III or IV sickle cell lung disease

Genotypically HLA identical sibling donor available

--Prior/Concurrent Therapy--

No prior transfusions with greater than 5 units RBC

--Patient Characteristics--

Performance status: Karnofsky 70-100%


  • No active hepatitis
  • No moderate/severe portal fibrosis

Renal: Glomerular filtration rate at least 30% predicted for age


  • No severe residual functional neurologic impairment
  • Hemiplegia alone allowed


  • HIV negative
  • Not pregnant or nursing
  • Fertile patients must use effective contraception

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): NCT00004485

United States, California
Children's Hospital of Oakland
Oakland, California, United States, 94609
United States, Washington
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Fred Hutchinson Cancer Research Center
Study Chair: Mark Walters Children's Hospital of Oakland Identifier: NCT00004485     History of Changes
Other Study ID Numbers: 199/14243
First Posted: October 19, 1999    Key Record Dates
Last Update Posted: September 10, 2008
Last Verified: September 2008

Keywords provided by Office of Rare Diseases (ORD):
genetic diseases and dysmorphic syndromes
hematologic disorders
rare disease
sickle cell anemia

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Mycophenolic Acid
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors
Antibiotics, Antineoplastic
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents