Summary: Chediak-Higashi syndrome (CHS) is a rare autosomal recessive disorder characterized in its classical form by oculocutaneous albinism, a bleeding diathesis, recurrent infection due to abnormal neutrophil and natural killer cell function, and eventual progression to a lymphohistiocytic infiltration known as the accelerated phase . Death often occurs within the first decade as a result of infection o...
Who is this study for: Child to young adult patients with Immune Function Disorders
Enrollment Status: Recruiting
Publish Date: April 27, 2022
Intervention Type: Drug
Study Drugs: AlemtuzumabFludarabineThiotepaMelphalan
Study Phase: Phase 2
Summary: This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.