Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

Severe Thalassemia Clinical Trial

— 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01005576
• Other study ID #: TCRN-NMD 0901
• Status: Completed
• Phase: Phase 2
• First received: October 29, 2009
• Last updated: November 17, 2017
• Start date: January 2010
• Est. completion date: July 2014

Study information

• Verified date: November 2017
• Source: Washington University School of Medicine
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: July 2014
• Est. primary completion date: April 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 16 Years

Eligibility

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

Related Conditions & MeSH terms

• Severe Thalassemia
• Thalassemia

Intervention

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

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory University	Atlanta	Georgia
United States	Dana Farber Cancer Institute	Boston	Massachusetts
United States	The University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	Children's Memorial Hospital	Chicago	Illinois
United States	The Research Institute at Nationwide Children's Hospital	Columbus	Ohio
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Duke University	Durham	North Carolina
United States	Regents of University of California- UCLA	Los Angeles	California
United States	University of Miami	Miami	Florida
United States	Vanderbilt University	Nashville	Tennessee
United States	Children's Hospital and Research Center at Oakland	Oakland	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Washington University	Saint Louis	Missouri
United States	All Children's Research Institute, Inc.	Saint Petersburg	Florida
United States	Methodist Healthcare System of San Antonio	San Antonio	Texas
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (3)

Lead Sponsor	Collaborator
Washington University School of Medicine	New England Research Institutes, Pediatric Blood and Marrow Transplant Consortium

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Primary Objective: Event-free Survival at 1 Year.		1 year
Secondary	Development of Graft Versus Host Disease (GVHD)		2 years
Secondary	Median Time to ANC Engraftment		100 days
Secondary	Median Time to Platelet Engraftment		100 days
Secondary	Incidence of Disease Recurrence		2 years