Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

This study has been completed.
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004485
First received: October 18, 1999
Last updated: September 8, 2008
Last verified: September 2008
  Purpose

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 Intervention Phase
Sickle Cell Anemia
Drug: cyclosporine
Drug: fludarabine
Drug: mycophenolate mofetil
Procedure: Bone Marrow Transplantation
Phase 1
Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 50
Study Start Date: December 1999
Primary Completion Date: August 2007 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   up to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

PROTOCOL ENTRY CRITERIA:

--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%

Hepatic:

  • No active hepatitis
  • No moderate/severe portal fibrosis

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

Neurologic:

  • No severe residual functional neurologic impairment
  • Hemiplegia alone allowed

Other:

  • HIV negative
  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004485

Locations
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
Investigators
Study Chair: Mark Walters Children's Hospital of Oakland
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00004485     History of Changes
Other Study ID Numbers: 199/14243, FHCRC-1373.00
Study First Received: October 18, 1999
Last Updated: September 8, 2008
Health Authority: Unspecified

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
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Cyclosporins
Cyclosporine
Mycophenolic Acid
Mycophenolate mofetil
Fludarabine monophosphate
Fludarabine
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Therapeutic Uses
Dermatologic Agents
Antirheumatic Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antimetabolites, Antineoplastic
Antimetabolites

ClinicalTrials.gov processed this record on July 22, 2014