New transplant approach aims to cure blood failure without harming lungs or liver
NCT ID NCT01659606
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk. About 40 participants will receive this gentler transplant to see if it leads to successful engraftment and survival.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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 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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a drug revive blood cell production in children with aplastic anemia?
- Could a platelet-boosting drug shorten recovery after stem cell transplants?
- A common antioxidant may help platelets recover faster after stem cell transplant
- Could a cancer antibody reboot blood production in aplastic anemia?
- Triple therapy aims to overcome antibody barrier in Half-Matched stem cell transplants
- New transplant method aims to reduce complications in bone marrow failure patients