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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01005576
Recruitment Status : Completed
First Posted : November 2, 2009
Results First Posted : October 20, 2017
Last Update Posted : December 13, 2017
New England Research Institutes
Pediatric Blood and Marrow Transplant Consortium
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Severe Thalassemia Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Intervention Model: Single Group Assignment
Masking: None (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)
Study Start Date : January 2010
Actual Primary Completion Date : April 2014
Actual Study Completion Date : July 2014

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. Primary Objective: Event-free Survival at 1 Year. [ Time Frame: 1 year ]

Secondary Outcome Measures :
  1. Development of Graft Versus Host Disease (GVHD) [ Time Frame: 2 years ]
  2. Median Time to ANC Engraftment [ Time Frame: 100 days ]
  3. Median Time to Platelet Engraftment [ Time Frame: 100 days ]
  4. Incidence of Disease Recurrence [ Time Frame: 2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01005576

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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.
Saint 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
Saint 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
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Principal Investigator: Shalini Shenoy, MD Washington University School of Medicine

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Responsible Party: Washington University School of Medicine Identifier: NCT01005576     History of Changes
Other Study ID Numbers: TCRN-NMD 0901
First Posted: November 2, 2009    Key Record Dates
Results First Posted: October 20, 2017
Last Update Posted: December 13, 2017
Last Verified: November 2017

Keywords provided by Washington University School of Medicine:
Hematopoietic cell transplant

Additional relevant MeSH terms:
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Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Fludarabine phosphate
Antineoplastic Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Antineoplastic Agents, Immunological