Sickle cell disease is a lifelong blood condition that can cause damage to the brain and other organs of the body. Children may develop severe clinical states, which includes stroke or abnormal blood flow to the brain. Treatment may include blood transfusions which may be required to prevent some of the conditions caused by this disease. Unfortunately, blood transfusions can cause iron overload, which can lead to severe and sometimes fatal complications.

Stem cells are young blood cells that can grow to make new blood cells such as white blood cells that help fight infections, platelets that help the blood to clot, and red blood cells that carry oxygen to the vital organs of the body. These cells may be taken from one individual (donor) and given to another (recipient). These stem cells, when placed in the body of the recipient, travel through the body to the bone marrow space and begin to grow and make new blood cells.

A stem cell transplant has been shown to help, and possibly cure, patients with sickle cell disease. Stem cells taken from a brother or sister may provide bone marrow that is a perfect match (same tissue type) for the recipient. Unfortunately, only about 10-20% of sickle cell patients have a matched sibling donor. Stem cells from partially matched (partial tissue match) family members have been tried with a few children with sickle cell disease. The risk and benefits of these types of transplants are not as well known as transplants using a matched donor. When children with sickle cell disease have no matched brother or sister donor, allogeneic transplants are a possible treatment available for these patients.

Condition	Intervention	Phase
Sickle Cell Disease	Procedure: Hematopoietic Stem Cell Transplantation	Phase I

Further Study Details: 

Primary Outcomes: To assess the safety of hematopoietic stem cell transplantation for children and adolescents with sickle cell disease.
Secondary Outcomes: 1. To determine the overall and disease-free survival 1 year after transplantation.; 2. To learn about how donor cells engraft (are accepted by the research participant’s body and begin to grow) and how these new cells work in research participants who receive this therapy.; 3. To learn about the rates of graft-versus-host disease after transplantation.; 4. To learn whether patients need additional stem cells or donor T-cells after transplantation.; 5. To find out if the damage already done by sickle cell disease to the brain can be helped by a stem cell transplant.; 6.To learn how the patient’s immune system recovers after transplantation.
Expected Total Enrollment:  6

Patients are evaluated to determine their eligibility for transplantation. Tests are done for various infections. Test of the heart, lungs, brain, and kidneys are done. The patient’s parents are also tested to make sure they are in good condition to donate blood stem cells and to determine who is the best tissue match. Patients who are enrolled receive a three month “pre-conditioning” treatment with two medicines to prevent rejection of the cells. These medicines are hydroxyurea and azathioprine. These medicines have been used successfully to decrease rejection in another disease.

After this 3 month period, patients then are admitted to the hospital to receive the transplant. The first part involves receiving chemotherapy and antibody treatments to weaken the immune system and to make space for the stem cells from the donor. The donor will donate stem cells twice: once at the beginning of the three month “pre-conditioning” period and once while the patient is receiving the chemotherapy in the hospital. The donor will receive shots under the skin for 6 days of a medication called G-CSF, which increases the number of stem cells in the blood stream. The stem cells are collected by placing an IV and using a pheresis machine on one or two consecutive days. The process takes 3-8 hours per day. Once the stem cells are collected, they are processed by a machine which removes the T-cells. These cells are then given to the patient. The T-cells are removed to decrease the risk of a serious complication known as Graft-Versus-Host Disease.

Patients are generally hospitalized for 3-4 weeks after the transplant and then seen frequently in the stem cell transplant clinic. Patients are generally able to return home approximately 100 days after the transplant.

Ages Eligible for Study:  2 Years   -   16 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• Diagnosis of hemoglobin SS or S-beta thalassemia sickle cell disease
• Age between two years to sixteen years
• Participant has a partially-matched family member with hemoglobin AA (normal) or hemoglobin AS (sickle trait)

Exclusion Criteria:

• Poor health (poor functional status)
• Active liver infection or scarring
• Severe kidney or heart problems
• Pregnancy or active breast feeding
• Antibodies against tha donor''''s tissue type due to transfusions
• No partially-matched family donor
• Severe lung problems related to sickle cell disease

Please refer to this study by ClinicalTrials.gov identifier  NCT00152113

Paul Woodard, M.D.      1-866-278-5833    info@stjude.org

Tennessee
      St. Jude Children''''s Research Hospital, Memphis,  Tennessee,  38105,  United States; Recruiting

Study chairs or principal investigators

Paul Woodard, M.D.,  Principal Investigator,  St. Jude Children''''s Research Hospital   

Study ID Numbers:  SCDHAP
Last Updated:  September 8, 2005
Record first received:  September 7, 2005
ClinicalTrials.gov Identifier:  NCT00152113
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-09-13