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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
New England Research Institutes
Pediatric Blood and Marrow Transplant Consortium
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT01005576
First received: October 29, 2009
Last updated: January 17, 2013
Last verified: January 2013
History of Changes
  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 2

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
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: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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
 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:   1 Year to 16 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01005576

Locations
United States, California
Regents of University of California- UCLA
Los Angeles, California, United States, 90095
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
University of Miami
Miami, Florida, United States, 33136
All Children's Research Institute, Inc.
St. Petersburg, Florida, United States, 33701
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Missouri
Washington University
St. Louis, Missouri, United States, 63110
United States, North Carolina
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Duke University
Durham, North Carolina, United States, 27710
United States, Ohio
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
United States, Texas
UT Southwestern Medical Center
Dallas, Texas, United States, 75390
Methodist Healthcare System of San Antonio
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 School of Medicine
ClinicalTrials.gov Identifier: NCT01005576     History of Changes
Other Study ID Numbers: TCRN-NMD 0901
Study First Received: October 29, 2009
Last Updated: January 17, 2013
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 May 22, 2013