Stem Cell Transplant for Hemoglobinopathy

Sickle Cell Disease Clinical Trial

Official title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism

Status Completed

Clinical Trial Summary

This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease.

Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.

Clinical Trial Description

Prior to transplantation, subjects will receive either:

Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI)

Or

Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte colony-stimulating factor (GSCF)

These drugs (and the radiation) are being given to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.

After stem cell transplantation, subjects will be given cyclosporine-A and mycophenolate (MMF)/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient. ;

Related Conditions & MeSH terms

• Anemia, Diamond-Blackfan
• Anemia, Sickle Cell
• Diamond-Blackfan Anemia
• Hemoglobinopathies
• Neutropenia
• Severe Congenital Neutropenia
• Shwachman-Diamond Syndrome
• Sickle Cell Disease
• Thalassemia

• NCT number: NCT00176852
• Study type: Interventional
• Source: Masonic Cancer Center, University of Minnesota
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: June 2002
• Completion date: January 2020