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 educated T cells, which fight
infection. Without successful treatment, patients with cDGA must remain in reverse isolation
to prevent infection and subsequent death.
Cultured thymus tissue with and without immunosuppression (drugs given before and after
implantation) has resulted in the development of good T cell function in subjects with
complete DiGeorge anomaly.
This expanded access study continues cultured thymus tissue safety and efficacy research for
the treatment of complete DiGeorge anomaly. Eligible participants receive cultured thymus
tissue. Immune function testing is continued for one year post-implantation.
Details
Lead Sponsor:
M. Louise Markert
Collaborators:
Enzyvant Therapeutics GmbH Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) National Institute of Allergy and Infectious Diseases (NIAID) National Institutes of Health (NIH)