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Clinical Trial NCT03619551

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)

  • ClinicalTrials.gov ID: NCT03619551
  • Recruiting participants (Starts Nov. 1, 2018)
  • Not accepting healthy volunteers
  • UTSW Principal Investigator: VICTOR MICHAEL AQUINO

summary

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.

objective

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.

If you are interested in this clinical trial, please contact Sophia Williams on the Children’s Health Research Team.Call 214-648-6554Email