Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

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.

NCT Number	NCT01659606
Lead Sponsor	Boston Children's Hospital Collaborators: Dana-Farber Cancer Institute, Children's Hospital Medical Center, Cincinnati, Fred Hutch/University of Washington/Seattle Children's Cancer Consortium, Baylor College of Medicine, Children's Hospital of Philadelphia, University of Wisconsin, Madison, Karolinska University Hospital, Hackensack Meridian Health, Oslo University Hospital, Children's Mercy Hospital Kansas City, University of Chicago, Massachusetts General Hospital
Conditions	Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
Enrollment	40 participants
Start Date	2012-07
Primary Completion	2026-12 (estimated)
Study Completion	2034-12 (estimated)
Updated on ClinicalTrials.gov	2026-03-27