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Clinical Trial Detail

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

Complete title: Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita/telomere disease

Research Study Number 9861
Principal Investigator Lauri Burroughs, MD
Phase II

Research Study Description

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.

Eligibility Criteria (must meet the following to participate in this study)

** For Eligibility information, please click on the "Look up trial at NIH" link above. **

Other eligibility criteria may apply.

Research Study Number 9861
Contact Seattle Cancer Care Alliance Intake Office
Telephone 800-804-8824 / 206-606-1024

Keywords: Anemia, Aplastic; Hematologic Malignancies; Pediatric Cancers, Miscellaneous; Anemia; Bone Marrow Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Mental Disorders; Pathologic Processes; Dyskeratosis Congenita; Hoyeraal Hreidarsson Syndrome; Revesz Syndrome; Genetic Diseases, X-Linked

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