Clinical Trial: Thymus Transplantation Dose in DiGeorge #932

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Dose Study of Thymus Transplantation in DiGeorge Anomaly, IND 9836, #932.1

Brief Summary: One purpose of this study is to determine whether the amount of thymus tissue transplanted into DiGeorge anomaly infants has any effect on the immune outcome. Another purpose of this study is to determine whether parental parathyroid gland transplantation (in addition to thymus transplantation) can help both the immune and the calcium problems in DiGeorge infants with hypocalcemia. [Funding Source - FDA OOPD]

Detailed Summary:

DiGeorge anomaly is a congenital disorder in which infants are born with defects of the thymus, heart, and parathyroid gland. Complete DiGeorge Anomaly is usually fatal within the first two years of life. This trial evaluates the role of thymus tissue dose in thymus transplantation in complete (typical) DiGeorge anomaly infants, and continues safety assessments.

DiGeorge infants who have successful thymus transplants but remain with hypoparathyroidism must go to the clinic for frequent calcium levels and to the hospital for calcium infusions; these infants are at risk for seizures from low calcium. Approximately ½ of infants with profound hypoparathyroidism will develop nephrocalcinosis. This protocol had a parental parathyroid transplant arm for complete DiGeorge infants with athymia and profound hypoparathyroidism.


Sponsor: M. Louise Markert

Current Primary Outcome: Regression analyses used to correlate dose to immunologic parameters: T cell proliferative response; naïve T cells; and T cell variability. [ Time Frame: 1 year post-transplantation ]

Original Primary Outcome: Regression analyses used to correlate dose to immunologic parameters: T cell proliferative response; naïve T cells; and T cell variability. The arms are also compared to determine if parathyroid transplants have adverse effects on immune outcomes. [ Time Frame: 1 year post-transplantation ]

Current Secondary Outcome:

  • Thymus transplantation efficacy: survival is recorded. Immune reconstitution efficacy: T cell phenotypic and functional parameters are evaluated. This is evaluated in descriptive fashion. [ Time Frame: Ongoing ]
  • Parental parathyroid transplantation efficacy: number of subjects who are off calcium and calcitriol supplementation. The time that calcium supplementation needs to be resumed is recorded. [ Time Frame: 1 year post-transplantation ]
  • Safety. Particular attention on oligoclonal T cell development; pulmonary complications; infections; and autoimmune diseases. Dose is correlated with number of subjects who get rashes lasting >1 week with development of wheezing or oxygen requirement. [ Time Frame: Ongoing - Post-Transplantation ]
  • Correlations between dose and other immune parameters and factors which might affect outcome including HLA matching and thymus donor heart defect. Evaluate whether HLA-DR matching results in increased total CD4 T cell numbers. [ Time Frame: 1 year post-transplantation & ongoing ]


Original Secondary Outcome:

  • Thymus transplantation efficacy: survival is recorded. Immune reconstitution efficacy: T cell phenotypic and functional parameters are evaluated. This will be evaluated in descriptive fashion. [ Time Frame: Ongoing ]
  • Parental parathyroid transplantation efficacy: number of subjects who are off calcium and calcitriol supplementation. The time that calcium supplementation needs to be resumed is recorded. [ Time Frame: 1 year post-transplantation ]
  • Safety. Particular attention on oligoclonal T cell development; pulmonary complications; infections; and autoimmune diseases. Dose is correlated with number of subjects who get rashes lasting >1 week with development of wheezing or oxygen requirement. [ Time Frame: Ongoing - Post-Transplantation ]
  • Correlations between dose and other immune parameters and factors which might affect outcome including HLA matching and thymus donor heart defect. Evaluate whether HLA-DR matching results in increased total CD4 T cell numbers. [ Time Frame: 1 year post-transplantation & ongoing ]


Information By: Duke University

Dates:
Date Received: December 17, 2007
Date Started: February 2006
Date Completion: June 2027
Last Updated: May 2, 2017
Last Verified: May 2017