MT2017-17:T Cell receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in patients with Inherited Bone Marrow Failure (BMF) Disorders
The purpose of this study is to learn if removing the donor T cells from the donor product using this new method will be a better way to reduce the risk of GVHD. The benefit of removing these cells with this new method is that they will prevent GVHD without requiring drugs to suppress the immune system. Potentially, the immune system will recover from the transplant faster, which in turn will also lessen the risk of severe infections. As well, the patient will not have the other common undesired side effects of these immunosuppressive drugs.
• up to 65 years of age
• have a diagnosis of Fanconi anemia
• have a suitable donor for peripheral blood cells
• women of childbearing potential and men with partners of child-bearing potential must agree to use of contraception for the duration of treatment and 4 months after the transplant
• see link to clinicaltrials.gov for additional criteria
• women who are pregnant or breastfeeding
• cancer within previous 2 years
Drug: Alemtuzumab, Drug: Busulfan, Drug: Cyclophosphamide (CY) (Plan 1), Drug: Cyclophosphamide (CY) (Plan 2), Device: Donor mobilized PBSC infusion, Drug: Fludarabine (FLU), Drug: G-CSF, Drug: Melphalan, Drug: Methylprednisolone (MP), Drug: Rituximab, Drug: Total Body Irradiation (TBI) (Plan 1)
Blood Disorders, Rare Diseases
Clinics and Surgery Center (CSC), Fanconi Anemia, Myelodysplastic Syndromes, Severe Aplastic Anemia