Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
This trial is active, not recruiting.
|Treatment||transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan|
|Sponsor||Washington University School of Medicine|
|Collaborator||New England Research Institutes|
|Start date||January 2010|
|End date||April 2014|
|Trial size||20 participants|
|Trial identifier||NCT01005576, TCRN-NMD 0901|
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.
|United States||No locations recruiting|
|Other Countries||No locations recruiting|
|Los Angeles, CA||Regents of University of California- UCLA||no longer recruiting|
|Oakland, CA||Children's Hospital and Research Center at Oakland||no longer recruiting|
|Washington, DC||Children's National Medical Center||no longer recruiting|
|Miami, FL||University of Miami||no longer recruiting|
|St. Petersburg, FL||All Children's Research Institute, Inc.||no longer recruiting|
|Atlanta, GA||Emory University||no longer recruiting|
|Chicago, IL||Children's Memorial Hospital||no longer recruiting|
|Boston, MA||Dana Farber Cancer Institute||no longer recruiting|
|Ann Arbor, MI||University of Michigan||no longer recruiting|
|St. Louis, MO||Washington University||no longer recruiting|
|Chapel Hill, NC||The University of North Carolina at Chapel Hill||no longer recruiting|
|Durham, NC||Duke University||no longer recruiting|
|Columbus, OH||The Research Institute at Nationwide Children's Hospital||no longer recruiting|
|Portland, OR||Oregon Health and Science University||no longer recruiting|
|Philadelphia, PA||The Children's Hospital of Philadelphia||no longer recruiting|
|Nashville, TN||Vanderbilt University||no longer recruiting|
|Dallas, TX||UT Southwestern Medical Center||no longer recruiting|
|San Antonio, TX||Methodist Healthcare System of San Antonio||no longer recruiting|
|Intervention model||single group assignment|
Primary objective: To determine event-free survival at 1 year.
time frame: 1 year
Secondary objectives: To determine the effect of hematopoietic cell transplant on clinical and laboratory manifestations of thalassemia and determining the incidence of transplant-related outcomes for 2 years.
time frame: 2 years
Male or female participants from 1 year up to 16 years old.
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
|Official title||A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)|
|Principal investigator||Shalini Shenoy, MD|
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