Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
- Conditions
- Severe Thalassemia
- Interventions
- Registration Number
- NCT01005576
- Lead Sponsor
- Washington University School of Medicine
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 21
- 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
- Pregnant or breastfeeding
- HIV positive
- Prior allogeneic marrow or stem cell transplantation
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Conditioning regimen Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0
- Primary Outcome Measures
Name Time Method Primary Objective: Event-free Survival at 1 Year. 1 year
- Secondary Outcome Measures
Name Time Method Median Time to ANC Engraftment 100 days Median Time to Platelet Engraftment 100 days Development of Graft Versus Host Disease (GVHD) 2 years Incidence of Disease Recurrence 2 years
Trial Locations
- Locations (18)
Children's Memorial Hospital
🇺🇸Chicago, Illinois, United States
All Children's Research Institute, Inc.
🇺🇸Saint Petersburg, Florida, United States
Children's Hospital and Research Center at Oakland
🇺🇸Oakland, California, United States
Regents of University of California- UCLA
🇺🇸Los Angeles, California, United States
The Research Institute at Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Methodist Healthcare System of San Antonio
🇺🇸San Antonio, Texas, United States
Emory University
🇺🇸Atlanta, Georgia, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
Dana Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Vanderbilt University
🇺🇸Nashville, Tennessee, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Duke University
🇺🇸Durham, North Carolina, United States
University of Miami
🇺🇸Miami, Florida, United States
The University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States