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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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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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 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