Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Status:
Recruiting
Trial end date:
2034-12-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital
Collaborators:
Baylor College of Medicine Children's Hospital Los Angeles Children's Hospital Medical Center, Cincinnati Children's Hospital of Philadelphia Children's Mercy Hospital Kansas City Dana-Farber Cancer Institute Duke University Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium Hackensack Meridian Health Karolinska University Hospital Massachusetts General Hospital Mayo Clinic Oslo University Hospital University of Chicago University of Wisconsin, Madison