Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

NCT00004485 | Completed Phase 1

• 2 other identifiers: 199/14243FHCRC-1373.00
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 2

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.

Conditions

Sickle Cell Anemia

Keywords

genetic diseases and dysmorphic syndromes; hematologic disorders; rare disease; sickle cell anemia

Interventions

cyclosporine DRUG

fludarabine DRUG

mycophenolate mofetil DRUG

Bone Marrow Transplantation PROCEDURE

Eligibility Criteria

• Age: Up to 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Fred Hutchinson Cancer Center: lead

Study Sites (2)

Children's Hospital of Oakland

Oakland, California, 94609, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Genetic Diseases, Inborn; Hematologic Diseases; Rare Diseases

Interventions

Cyclosporine; fludarabine; Mycophenolic Acid; Bone Marrow Transplantation

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemic and Lymphatic Diseases; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids; Tissue Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative

Study Officials

• Mark Walters

  Children's Hospital of Oakland

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: December 1, 1999
• Primary Completion: August 1, 2007
• Last Updated: September 10, 2008

Record last verified: 2008-09

Locations

US (2)