This is a single-arm, open label, phase 2 study to determine the safety and efficacy of vorinostat without serotherapy as GVHD prophylaxis when combined with either tacrolimus and methotrexate or post-transplant cyclophosphamide, tacrolimus, and mycophenolate in patients aged 1 to 26 years of age with non-malignant disorders undergoing bone marrow transplant following myeloablative conditioning.
Primary Immunodeficiency/Primary Immune regulatory disorders
Inborn errors of metabolism
Red blood cell disorders including hemoglobinopathies per protocol.
Inherited bone marrow failure syndromes
Diagnosis of idiopathic severe aplastic anemia
Diagnosis of severe combined immunodeficiency syndrome
Diagnosis of malignancy within the last 5 years.
Diagnosis of Epstein-Barr virus (EBV)-driven lymphoproliferative disorder within the last 5 years
Sung Won Choi
Lead sponsor
University of Michigan
Sponsor institution