Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

• Status: Completed
• Trial end date: 2014-07-01
• Target enrollment: 0
• Participant gender: All

Summary

This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.

This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.

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

Details

• Lead Sponsor: Washington University School of Medicine
• Collaborators: HealthCore-NERI; New England Research Institutes; Pediatric Blood and Marrow Transplant Consortium
• Treatments: Alemtuzumab; Fludarabine; Fludarabine phosphate; Melphalan

Criteria

Inclusion Criteria:

• 1-16.00 years old

• Have transfusion dependent thalassemia major

• Shall not have an HLA-matched family donor

• Must have a suitably matched unrelated marrow donor or UCB product

• Lansky score >/= 70

• Adequate pulmonary, renal, liver, and other organ function as defined in protocol

• Negative pregnancy test

• Adequate total nucleated cell or CD34+ dose of product as defined in protocol

• Iron chelation must be discontinued >/= 48 hours prior to conditioning regimen

Exclusion Criteria:

• Pregnant or breastfeeding

• HIV positive

• Prior allogeneic marrow or stem cell transplantation