PBMTC NMD 1801 (CSIDE), A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC “CSIDE” Protocol (Conditioning SCID Infants Diagnosed Early)
Study ID: STU-2018-0210
This is a prospective, multicenter, phase ii, open-label study of two reduced busulfan dose levels in newborns identified at birth with SCiD of appropriate genotype/phenotype and clinical status, undergoing either haploidentical related or well-matched unrelated donor TCR[RegisteredTM][MiCRo-SYMBoL]+/CD19+ depleted HCT. Subjects will be enrolled on either of 2 strata according to genotype (defects of cytokine receptor function i.e. iL2RG or JaK3 and defects of receptor recombination i.e. RaG1 or RaG2). Thus up to 32 subjects on each of 2 strata or 64 subjects total would be enrolled over 4 years with 3 years follow-up.
To evaluate the safety and efficacy of TCR[RegisteredTM][MiCRo-SYMBoL]+/CD19+ depleted allogeneic HCT following either a low dose or moderate dose busulfan-based reduced intensity conditioning regimen in the treatment of 2 genotype-driven cohorts of infants diagnosed at birth with SCiD.