Clinical trial NCT01005576: Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Thalassemia
Keywords: Thalassemia
Alemtuzumab
Hematopoietic cell transplant
non-myeloablative
Lead sponsor: Washington University School of Medicine
Sponsor class: OTHER
Collaborators: name: Carelon Research
name: Pediatric Blood and Marrow Transplant Consortium
First posted: 2009-11-01
Enrollment: 21
Sex: ALL
Minimum age (years): 1
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Primary Objective: Event-free Survival at 1 Year.
timeFrame: 1 year
Secondary outcomes:
measure: Development of Graft Versus Host Disease (GVHD)
timeFrame: 2 years
measure: Median Time to ANC Engraftment
timeFrame: 100 days
measure: Median Time to Platelet Engraftment
timeFrame: 100 days
measure: Incidence of Disease Recurrence
timeFrame: 2 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Regents of University of California- UCLA
state: California
city: Oakland
country: United States
facility: Children's Hospital and Research Center at Oakland
state: California
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Saint Petersburg
country: United States
facility: All Children's Research Institute, Inc.
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Columbus
country: United States
facility: The Research Institute at Nationwide Children's Hospital
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: San Antonio
country: United States
facility: Methodist Healthcare System of San Antonio
state: Texas
Study officials:
affiliation: Washington University School of Medicine
name: Shalini Shenoy, MD
role: PRINCIPAL_INVESTIGATOR
References: