Clinical Trial: Thymus Transplantation in DiGeorge Syndrome #668
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: Phase II Study of Thymus Transplantation in Complete DiGeorge Syndrome #668
Brief Summary: The study purpose is to determine whether thymus transplantation without immunosuppression is effective in treating typical complete DiGeorge syndrome.
Detailed Summary:
There is no safe and effective treatment for DiGeorge syndrome and most patients die by the age of two. Complete DiGeorge syndrome is characterized by very low T cell or very low naïve T cell numbers. In this study, typical complete DiGeorge syndrome subjects received human postnatal cultured thymus tissue transplants. Thymus tissue that would otherwise be discarded was transplanted into DiGeorge subjects in the operating room. At the time of transplantation, a skin biopsy was obtained to look for any preexisting T cells. After transplantation, subjects were followed by routine research immune evaluations, using blood samples obtained every 2-4 weeks. At approximately 2-3 months post-transplantation subjects underwent an open biopsy of the allograft. The biopsy was done under general anesthesia in the operating room. At the time of the graft biopsy, another skin biopsy was obtained to look for clonal populations of T cells.
The protocol aims include: assessing thymopoiesis in the allograft biopsy; assessing immunoreconstitution of complete DiGeorge syndrome subjects after postnatal allogeneic thymus transplantation; assessing minimally invasive methods of assessing thymopoiesis (flow cytometry and polymerase chain reaction (PCR); assessing pre-transplant T cells which do not proliferate in response to mitogens (focusing on NK-T cells); and, assessing thymus transplantation safety and toxicity.
Sponsor: M. Louise Markert
Current Primary Outcome: Survival rate at one year post-transplantation. [ Time Frame: One year post-transplantation. ]
Original Primary Outcome: Survival rate at one year post-transplantation. T cell reconstitution: proliferative response to Tetanus Toxoid of greater than ten-fold. [ Time Frame: One year post-transplantation. ]
Current Secondary Outcome: T cell proliferative response to tetanus toxoid [ Time Frame: Approximately 1 year after transplantation ]
Original Secondary Outcome: T cell responses to mitogens and T cell numbers [ Time Frame: Ongoing ]
Information By: Duke University
Dates:
Date Received: December 17, 2007
Date Started: November 2001
Date Completion: June 2027
Last Updated: September 22, 2016
Last Verified: September 2016
