Breakthrough transplant method aims to cure blood disease without toxic side effects
NCT ID NCT01659606
Summary
This study is testing a new, gentler bone marrow transplant procedure for people with dyskeratosis congenita, a rare genetic disorder that causes bone marrow failure. The standard transplant uses harsh drugs and radiation that can worsen lung/liver disease and cancer risk in these patients. This trial aims to see if avoiding those damaging agents can still successfully cure the blood disease while improving long-term safety and survival.
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Contacts and locations
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Locations
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Boston Children's Hospital (pediatric patients)
Boston, Massachusetts, 02115, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Mercy Hospital Kansas City
Kansas City, Missouri, 64108, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Dana-Farber Cancer Institute (adult patients)
Boston, Massachusetts, 02115, United States
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Fred Hutch/University of Washington/Seattle Children's Cancer Consortium
Seattle, Washington, 98109, United States
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Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
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Karolinska University Hospital
Stockholm, Sweden
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Oslo University Hospital
Oslo, Norway
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University of Chicago
Chicago, Illinois, 60637, United States
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University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792, United States
Conditions
Explore the condition pages connected to this study.