Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

• Status: Completed
• Trial end date: 2009-07-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies bone marrow and restore circulating blood counts. In addition the incidence and severity of side effects and of graft vs. host disease (GVHD) will be monitored.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dana-Farber Cancer Institute
• Collaborators: Beth Israel Deaconess Medical Center; Brigham and Women's Hospital; Emory University; Feist-Weiller Cancer Center at Louisiana State University Health Sciences; Massachusetts General Hospital; Ohio State University
• Treatments: Alemtuzumab; Busulfan; Fludarabine; Fludarabine phosphate

Criteria

Inclusion Criteria:

• Patients with sickle cell disease should have one or more of the following: acute chest syndrome requiring hospitalization; nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours; recurrent caso-occlusive pain or recurrent priapism; sickle neuropathy; bilateral proliferative retinopathy and major visual impairment of at least one eye; osteonecrosis of multiple joints; transfusion dependence; vaso-occlusive.

• Patients with thalassemia should have one or more of the following: transfusion dependence; iron overload; presence of 2 or more alloantibodies against red cell antigens.

Exclusion Criteria:

• Pregnancy

• Acute hepatitis

• Cardiac ejection fraction < 30%

• Severe renal impairment

• Severe residual functional neurologic impairment

• Evidence of HIV infection