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Status: RecruitingPhase: Phase 2Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)NCT ID: NCT01659606
(View complete trial on ClinicalTrials.gov)
DFCI Protocol ID:
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease and a predisposition to cancer are also frequent causes of illness and death. Bone marrow transplantation (BMT) can cure the blood system but can make the lung disease and cancer predisposition worse, because of 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 agents in patients with DC, and still have a successful BMT. In this protocol we will test whether a regimen that avoids alkylators and radiation can permit successful BMT without compromising survival in patients with DC.
Conducting Institutions: Dana-Farber Cancer Institute, Children's Hospital Boston
Overall PI: Suneet Agarwal, MD, PhD,
Children's Hospital Boston
Contacts: Dana-Farber Cancer Institute:
Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP,