Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
NCT ID: NCT01005576
Last Updated: 2017-12-13
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
21 participants
INTERVENTIONAL
2010-01-31
2014-07-31
Brief Summary
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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
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Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Conditioning regimen
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
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
Interventions
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Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
Eligibility Criteria
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Inclusion Criteria
* 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
* HIV positive
* Prior allogeneic marrow or stem cell transplantation
1 Year
16 Years
ALL
No
Sponsors
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Carelon Research
OTHER
Pediatric Blood and Marrow Transplant Consortium
OTHER
Washington University School of Medicine
OTHER
Responsible Party
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Principal Investigators
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Shalini Shenoy, MD
Role: PRINCIPAL_INVESTIGATOR
Washington University School of Medicine
Locations
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Regents of University of California- UCLA
Los Angeles, California, United States
Children's Hospital and Research Center at Oakland
Oakland, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Miami
Miami, Florida, United States
All Children's Research Institute, Inc.
St. Petersburg, Florida, United States
Emory University
Atlanta, Georgia, United States
Children's Memorial Hospital
Chicago, Illinois, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Washington University
St Louis, Missouri, United States
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Duke University
Durham, North Carolina, United States
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Vanderbilt University
Nashville, Tennessee, United States
UT Southwestern Medical Center
Dallas, Texas, United States
Methodist Healthcare System of San Antonio
San Antonio, Texas, United States
Countries
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Other Identifiers
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TCRN-NMD 0901
Identifier Type: -
Identifier Source: org_study_id