Serum-Free Thymus Transplantation in DiGeorge Anomaly
- Conditions
- DiGeorge Anomaly
- Interventions
- Biological: Serum Free Thymus Transplantation with ImmunosuppressionOther: Serum Free Thymus Transplantation without immunosuppression
- Registration Number
- NCT00849888
- Lead Sponsor
- Sumitomo Pharma Switzerland GmbH
- Brief Summary
The study purpose is to determine if thymus tissue cultured in a serum-free (SF) solution is a safe and effective treatment for atypical and typical complete DiGeorge anomaly. \[Funding Source - FDA OOPD\]
- Detailed Description
Complete DiGeorge anomaly is a congenital disorder characterized by athymia. Without successful treatment, patients remain immunodeficient and usually die by age 2 years. In "typical" complete DiGeorge subjects who have no T cells, thymus transplantation without immunosuppression has resulted in diverse T cell development and good T cell function. In "atypical" complete DiGeorge subjects who have no thymus, a rash, and some T cells that presumably developed extrathymically, thymus transplantation with immunosuppression has resulted in diverse T cell development and good T cell function. Thus far, thymus transplantation studies have used thymus cultured in fetal bovine serum (FBS medium). This protocol's purpose is to determine whether transplanted thymus cultured in serum free medium can safely support thymopoiesis and T cell reconstitution as does FBS medium cultured thymus tissue in DiGeorge anomaly subjects. This protocol includes 2 arms: atypical DiGeorge subjects who will receive immunosuppression and thymus transplantation; and, typical complete DiGeorge subjects who will receive thymus transplantation without immunosuppression. Serum free medium use would reduce concerns of animal product exposure including potential exposure to bovine spongiform encephalopathy(BSE).
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 2
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Atypical Complete DiGeorge Serum Free Thymus Transplantation with Immunosuppression Thymus Transplantation with Immunosuppression Typical Complete DiGeorge Serum Free Thymus Transplantation without immunosuppression Thymus Transplantation without Immunosuppression
- Primary Outcome Measures
Name Time Method Thymopoiesis or graft rejection on biopsy. Two months post-thymus transplantation. Graft rejection analysis by biopsy at 2 months post-thymus transplantation.
Incidence of graft-versus-host-disease (GVHD). One year post-thymus-transplantation. Development of graft versus host disease in first year after transplantation associated with T cells from the thymus donor.
Survival One year post-thymus transplantation. Survival at one year post thymus transplantation.
- Secondary Outcome Measures
Name Time Method Immune outcomes: T cell development; evaluate T cell numbers, diversity, and function. One year post-thymus transplantation. Number of naïve CD4 T cells at one year after transplantation Number of total CD4 T cells at one year after transplantation Proliferative response to PHA at one year after transplantation TCRBV diversity by spectratyping measured by DKL score at one year after transplantation
Incidence of autoimmune disease. By two years post-thymus transplantation. Incidence of autoimmune disease by year 2 after transplantation Cytopenias as assessed by complete blood counts and differential. Thyroid disease as assessed by thyroid function tests