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Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Sponsor: Boston Children's Hospital
Summary
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of illness and death. Bone marrow transplantation (BMT) can cure the blood system but can make the lung and liver disease and risk of cancer worse, because of DNA damaging agents such as alkylators and radiation that are typically used in the procedure. Based on the biology of DC, we hypothesize that it may be possible to avoid these DNA damaging agents in patients with DC, and still have a successful BMT. In this protocol we will test whether a regimen that avoids DNA alkylators and radiation can permit successful BMT without compromising survival in patients with DC.
Official title: Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease
Key Details
Gender
All
Age Range
30 Days - 65 Years
Study Type
INTERVENTIONAL
Enrollment
40
Start Date
2012-07
Completion Date
2034-12
Last Updated
2026-03-27
Healthy Volunteers
No
Interventions
alemtuzumab
Conditioning: alemtuzumab 0.2 mg/kg/dose IV/SC x 5 doses
Fludarabine
fludarabine 30 mg/m2/dose IV x 6 doses
Cyclosporins
Mycophenolate mofetil
Tacrolimus
Locations (13)
University of Chicago
Chicago, Illinois, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Boston Children's Hospital (pediatric patients)
Boston, Massachusetts, United States
Dana-Farber Cancer Institute (adult patients)
Boston, Massachusetts, United States
Children's Mercy Hospital Kansas City
Kansas City, Missouri, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Baylor College of Medicine
Houston, Texas, United States
Fred Hutch/University of Washington/Seattle Children's Cancer Consortium
Seattle, Washington, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States
Oslo University Hospital
Oslo, Norway
Karolinska University Hospital
Stockholm, Sweden