Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
| Status: | Completed |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 1 - 16 |
| Updated: | 12/15/2017 |
| Start Date: | January 2010 |
| End Date: | July 2014 |
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.
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.
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
We found this trial at
18
sites
Duke University Younger than most other prestigious U.S. research universities, Duke University consistently ranks among...
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Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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University of Miami A private research university with more than 15,000 students from around the...
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747 52nd St
Oakland, California 94609
Oakland, California 94609
(510) 428-3000
Children's Hospital and Research Center Oakland For nearly 100 years, Children's Hospital & Research Center...
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
Portland, Oregon 97239
503 494-8311
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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University of Michigan The University of Michigan was founded in 1817 as one of the...
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Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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U.T. Southwestern Medical Center The story of UT Southwestern Medical Center is one of commitment...
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Vanderbilt University Vanderbilt offers undergraduate programs in the liberal arts and sciences, engineering, music, education...
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111 Michigan Ave NW
Washington, District of Columbia
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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