Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)
Status and phase
Conditions
Treatments
Details and patient eligibility
About
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.
Enrollment
Sex
Ages
Volunteers
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
Trial design
Primary purpose
Allocation
Interventional model
Masking
21 participants in 1 patient group
Trial contacts and locations
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Data sourced from clinicaltrials.gov
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