Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)
This study is currently recruiting participants.
Verified on March 2011 by Washington University School of Medicine
First Received on October 29, 2009. Last Updated on March 2, 2011 History of Changes
Sponsor: Washington University School of Medicine
Collaborators: New England Research Institutes
Pediatric Blood and Marrow Transplant Consortium
Information provided by: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01005576
Purpose
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.
Condition Intervention Phase
Severe Thalassemia
Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Phase II
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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)
Resource links provided by NLM:
MedlinePlus related topics: Thalassemia
Drug Information available for: Fludarabine Fludarabine monophosphate Campath Alemtuzumab Melphalan Sarcolysin Melphalan hydrochloride
U.S. FDA Resources
Further study details as provided by Washington University School of Medicine:
Primary Outcome Measures:
Primary objective: To determine event-free survival at 1 year. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
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 ] [ Designated as safety issue: No ]
Estimated Enrollment: 20
Study Start Date: January 2010
Estimated Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Intervention Details:
Drug: 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
Ages Eligible for Study: 2 Years to 16 Years
Genders Eligible for Study: Both
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria:
2-16.00 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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01005576
Contacts
Contact: Lisa M Murray, MA, CCRP 314-454-4240 Murray_L@kids.wustl.edu
Locations
United States, California
Regents of University of California- UCLA Recruiting
Los Angeles, California, United States, 90095
Children's Hospital and Research Center at Oakland Recruiting
Oakland, California, United States, 94609
United States, Florida
All Children's Research Institute, Inc. Recruiting
St. Petersburg, Florida, United States, 33701
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
United States, Illinois
Children's Memorial Hospital Recruiting
Chicago, Illinois, United States, 60614
United States, Missouri
Washington University Recruiting
St. Louis, Missouri, United States, 63110
Contact: Lisa Murray, MA, CCRP 314-454-4240 Murray_L@kids.wustl.edu
Principal Investigator: Shalini Shenoy, MD
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27710
United States, Ohio
The Research Institute at Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Texas
Methodist Healthcare System of San Antonio Recruiting
San Antonio, Texas, United States, 78229
Sponsors and Collaborators
Washington University School of Medicine
New England Research Institutes
Pediatric Blood and Marrow Transplant Consortium
Investigators
Principal Investigator: Shalini Shenoy, MD Washington University Medical Center
More Information
No publications provided
Responsible Party: Washington University Medical Center ( Shalini Shenoy, MD )
ClinicalTrials.gov Identifier: NCT01005576 History of Changes
Other Study ID Numbers: TCRN-NMD 0901
Study First Received: October 29, 2009
Last Updated: March 2, 2011
Health Authority: United States: Institutional Review Board
Keywords provided by Washington University School of Medicine:
Thalassemia
Alemtuzumab
Hematopoietic cell transplant
non-myeloablative
Additional relevant MeSH terms:
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Melphalan
Fludarabine
Fludarabine monophosphate
Alemtuzumab
Campath 1G
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antineoplastic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antimetabolites, Antineoplastic
Antimetabolites
ClinicalTrials.gov processed this record on April 20, 2011