Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
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.
Timeline
- Start
- 2012-07
- Primary completion
- 2026-12
- Completion
- 2034-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Alemtuzumab | Monoclonal antibody | 0.2 mg/kg | Intravenous |
| Subject | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Background | Cyclosporine | Peptide | — | — |
| Background | Mycophenolate Mofetil | Small molecule | — | — |
| Background | Tacrolimus | Small molecule | — | — |