Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

November 17, 2017 updated by: Washington University School of Medicine

A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

21

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • Regents of University of California- UCLA
      • Oakland, California, United States, 94609
        • Children's Hospital and Research Center at Oakland
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami
      • Saint Petersburg, Florida, United States, 33701
        • All Children's Research Institute, Inc.
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Children's Memorial Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Dana Farber Cancer Institute
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • The University of North Carolina at Chapel Hill
      • Durham, North Carolina, United States, 27710
        • Duke University
    • Ohio
      • Columbus, Ohio, United States, 43205
        • The Research Institute at Nationwide Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University
    • Texas
      • Dallas, Texas, United States, 75390
        • UT Southwestern Medical Center
      • San Antonio, Texas, United States, 78229
        • Methodist Healthcare System of San Antonio

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 16 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Primary Objective: Event-free Survival at 1 Year.
Time Frame: 1 year
1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
Development of Graft Versus Host Disease (GVHD)
Time Frame: 2 years
2 years
Median Time to ANC Engraftment
Time Frame: 100 days
100 days
Median Time to Platelet Engraftment
Time Frame: 100 days
100 days
Incidence of Disease Recurrence
Time Frame: 2 years
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Washington University School of Medicine

Collaborators

HealthCore-NERI
Pediatric Blood and Marrow Transplant Consortium

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2010

Primary Completion (Actual)

April 1, 2014

Study Completion (Actual)

July 1, 2014

Study Registration Dates

First Submitted

October 29, 2009

First Submitted That Met QC Criteria

October 29, 2009

First Posted (Estimate)

November 1, 2009

Study Record Updates

Last Update Posted (Actual)

December 13, 2017

Last Update Submitted That Met QC Criteria

November 17, 2017

Last Verified

November 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TCRN-NMD 0901

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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