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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.
 
ClinicalTrials.gov Identifier: NCT01005576
Recruitment Status : Completed
First Posted : November 1, 2009
Results First Posted : October 20, 2017
Last Update Posted : December 13, 2017
Sponsor:
Collaborators:
HealthCore-NERI
Pediatric Blood and Marrow Transplant Consortium
Information provided by (Responsible Party):
Washington University School of Medicine

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: N/A
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

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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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 ]

Eligibility Criteria
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Ages Eligible for Study:   1 Year to 16 Years   (Child)
Sexes Eligible for Study:   All
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
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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 ClinicalTrials.gov identifier (NCT number): NCT01005576


Locations
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Sponsors and Collaborators
Washington University School of Medicine
HealthCore-NERI
Pediatric Blood and Marrow Transplant Consortium
Investigators
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Principal Investigator: Shalini Shenoy, MD Washington University School of Medicine
More Information
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Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01005576    
Other Study ID Numbers: TCRN-NMD 0901
First Posted: November 1, 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:
Thalassemia
Alemtuzumab
Hematopoietic cell transplant
non-myeloablative
Additional relevant MeSH terms:
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Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Fludarabine
Melphalan
Alemtuzumab
 Antineoplastic Agents
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Antineoplastic Agents, Immunological