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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

Primary Purpose

Severe Thalassemia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Sponsored by
Washington University School of Medicine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Severe Thalassemia focused on measuring Thalassemia, Alemtuzumab, Hematopoietic cell transplant, non-myeloablative

Eligibility Criteria

1 Year - 16 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Regents of University of California- UCLA
  • Children's Hospital and Research Center at Oakland
  • Children's National Medical Center
  • University of Miami
  • All Children's Research Institute, Inc.
  • Emory University
  • Children's Memorial Hospital
  • Dana Farber Cancer Institute
  • University of Michigan
  • Washington University
  • The University of North Carolina at Chapel Hill
  • Duke University
  • The Research Institute at Nationwide Children's Hospital
  • Oregon Health and Science University
  • The Children's Hospital of Philadelphia
  • Vanderbilt University
  • UT Southwestern Medical Center
  • Methodist Healthcare System of San Antonio

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Conditioning regimen

Arm Description

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

Outcomes

Primary Outcome Measures

Primary Objective: Event-free Survival at 1 Year.

Secondary Outcome Measures

Development of Graft Versus Host Disease (GVHD)
Median Time to ANC Engraftment
Median Time to Platelet Engraftment
Incidence of Disease Recurrence

Full Information

First Posted
October 29, 2009
Last Updated
November 17, 2017
Sponsor
Washington University School of Medicine
Collaborators
Carelon Research, Pediatric Blood and Marrow Transplant Consortium
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1. Study Identification

Unique Protocol Identification Number
NCT01005576
Brief Title
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Acronym
URTH
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 Type
Interventional

2. Study Status

Record Verification Date
November 2017
Overall Recruitment Status
Completed
Study Start Date
January 2010 (undefined)
Primary Completion Date
April 2014 (Actual)
Study Completion Date
July 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Washington University School of Medicine
Collaborators
Carelon Research, Pediatric Blood and Marrow Transplant Consortium

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Thalassemia
Keywords
Thalassemia, Alemtuzumab, Hematopoietic cell transplant, non-myeloablative

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Conditioning regimen
Arm Type
Experimental
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Intervention Description
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 Measure Information:
Title
Primary Objective: Event-free Survival at 1 Year.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Development of Graft Versus Host Disease (GVHD)
Time Frame
2 years
Title
Median Time to ANC Engraftment
Time Frame
100 days
Title
Median Time to Platelet Engraftment
Time Frame
100 days
Title
Incidence of Disease Recurrence
Time Frame
2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shalini Shenoy, MD
Organizational Affiliation
Washington University School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Regents of University of California- UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Children's Hospital and Research Center at Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
All Children's Research Institute, Inc.
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Children's Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
Dana Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
The University of North Carolina at Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Duke University
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
The Research Institute at Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Methodist Healthcare System of San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States

12. IPD Sharing Statement

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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

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