Study Status: Available
Recruit Status: Available
Study Type: Expanded Access: Available for Intermediate-size Population, Treatment IND/Protocol
Official Title: Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836
Brief Summary:
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into T cells, which fight infection. Complete DiGeorge anomaly patients cannot fight infection and are immunodeficient. Without successful treatment, cDGA patients usually die by age 2 years.
Thymus transplantation with and without immunosuppression (drugs given before and after transplantation) has resulted in the development good T cell function in complete DiGeorge anomaly subjects.
This Phase I/II study continues thymus transplantation safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants undergo thymus transplantation and biopsy. Immune function testing is continued for one year post-transplantation.