T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients with Severe Sickle Cell Disease SCD and Other Transfusion-Dependent Anemias (NCT03653338)

The trial, T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients with Severe Sickle Cell Disease SCD and Other Transfusion-Dependent Anemias, will include T and B-cell depletion of an HLA-mismatched graft, obtained from mobilized blood of healthy, unrelated donors.

The reduced intensity regimen paired with immune-ablative conditioning is expected to preserve fertility, prevent severe GVHD, and minimize the possibility of immune rejection in patients with sickle cell disease and other transfusion-dependent anemias such as beta-thalassemia or Diamond-Blackfan anemia.

The protocol targets full or high grade mixed donor engraftment to facilitate resolution of disease-related symptoms and improve quality of life.