This phase II trial studies how well a reduced intensity conditioning regimen works in treating patients with non-cancer (non-malignant) disorders. Reduced intensity conditioning involves giving medicines that suppress the immune system before giving the donor stem cells. It does not completely eliminate the blood making cells in bone marrow. Giving a reduced intensity conditioning regimen may result in fewer short and long term side effects, shorter hospital stay than after conditioning that completely destroys the bone marrow, and blood counts may recover more quickly.
Additional locations may be listed on ClinicalTrials.gov for NCT01050855.
Locations matching your search criteria
United States
Pennsylvania
Philadelphia
Children's Hospital of PhiladelphiaStatus: Active
Contact: Timothy Steven Olson
Phone: 267-426-5516
PRIMARY OBJECTIVES:
I. Evaluate engraftment of patients with non-malignant disorders using a reduced-intensity conditioning regimen.
II. Evaluate event free survival.
OUTLINE: Patients are assigned to 1 of 4 arms.
ARM I (Age > 6 months with sickle cell disease or immune dysregulation, polyendocrinopathy, and enteropathy X-linked syndrome [IPEX]): Patients receive alemtuzumab intravenously (IV) over 2 hours on days -22 to -19, fludarabine IV on days -7 to -3, melphalan IV on day -2, cyclosporine IV on day -1, and then undergo stem cell infusion on day 0 in the absence of disease progression or unacceptable toxicity. Patients then receive mycophenolate mofetil orally (PO) three times daily (TID) on days 1-45 in the absence of disease progression or unacceptable toxicity.
ARM II (Age > 6 months with thalassemia major): Patients receive hydroxyurea PO daily on days -50 to -22, alemtuzumab IV over 2 hours on days -22 to -19, fludarabine IV on days -7 to -3, thiotepa IV on day -3, melphalan IV on day -2, cyclosporine IV on day -1, and then undergo stem cell infusion on day 0 in the absence of disease progression or unacceptable toxicity. Patients then receive mycophenolate mofetil PO TID on days 1-45 in the absence of disease progression or unacceptable toxicity.
ARM III (Age > 6 months with immune deficiencies or hemophagocytic disorders): Patients receive alemtuzumab subcutaneously (SC) or IV on days -14 to -10, fludarabine IV on days -7 to -3, melphalan IV on day -2, cyclosporine IV on day -1, and then undergo stem cell infusion on day 0 in the absence of disease progression or unacceptable toxicity. Patients then receive mycophenolate mofetil PO TID on days 1-45 in the absence of disease progression or unacceptable toxicity.
ARM IV (Age < 6 months): Patients receive alemtuzumab SC or IV on days -8 to -4, busulfan IV on days -6 and -5, fludarabine on days -6 to -2, cyclosporine IV on day -2, and then undergo stem cell infusion on day 0 in the absence of disease progression or unacceptable toxicity. Patients then receive mycophenolate mofetil PO TID on days 1-45 in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for up to 2 years.
Lead OrganizationChildren's Hospital of Philadelphia
Principal InvestigatorTimothy Steven Olson
