Thymus Transplantation Safety-Efficacy
Complete DiGeorge Anomaly, DiGeorge Syndrome, DiGeorge Anomaly
About this trial
This is an expanded access trial for Complete DiGeorge Anomaly focused on measuring DiGeorge Anomaly, Thymus Transplantation, DiGeorge Syndrome, Athymia, Low T cell numbers, Immunoreconstitution, Immunodeficiency, Complete DiGeorge, Typical DiGeorge, Atypical DiGeorge, Complete DiGeorge Anomaly, Cultured Thymus Tissue Implantation, Cultured Thymus Tissue, Congenital Athymia
Eligibility Criteria
Inclusion criteria for implantation of cultured thymus tissue: Must have 1 of following: 22q11.2ds or 10p13 hemizygosity; hypocalcemia requiring replacement; congenital heart disease; or CHARGE syndrome or CHD7 mutation Complete DiGeorge: <50 CD3+ T cells/cumm or <50 CD3+ T cells/cumm that are CD62L+ CD45RA+, or <5% of CD3+ cells are CD62L+ CD45RA+ Atypical DiGeorge subjects must have, or have had, a rash. Group 1 •Typical cDGA whose T cells have a phytohemagglutinin (PHA) response of < 5,000 counts per minute (cpm) and < 20 fold PHA response. Group 2 •Typical cDGA whose T cells have a PHA response of >5,000 cpm and <50,000 cpm and >20 fold PHA response. Group 3 Typical cDGA whose T cells have a PHA response of >50,000 cpm. Typical cDGA with maternal engraftment Atypical cDGA whose T cells have a PHA response of <40,000 cpm when on immunosuppression or <75,000 cpm to PHA when not on immunosuppression. Atypical cDGA with group 3 PHA response & maternal engraftment Group 4 Atypical cDGA with PHA responses of >75,000 cpm while on no immunosuppression or a PHA responses of >40,000 cpm while on immunosuppression. Atypical cDGA with maternal engraftment and group 4 PHA response Exclusion criteria for implantation of cultured thymus tissue: Heart surgery conducted less than 4 weeks prior to projected implantation date. Heart surgery anticipated within 3 months after the proposed time of implantation Rejection by surgeon or anesthesiologist as surgical candidate Lack of sufficient muscle tissue to accept a transplant HIV infection Prior attempts at immune reconstitution, such as bone marrow transplant or previous thymus transplant CMV infection: For Groups 2, 3, and 4 CMV infection documented by >500 copies/ml in the blood by PCR on two consecutive assays. Ventilator Dependence or Positive Pressure Support: Ventilator support or positive pressure support, such as Continuous Positive Airway Pressure (CPAP) or Bi-level Positive Airway Pressure (BiPAP) support for a condition that is deemed to be severe or irreversible or which renders the subject too clinically unstable to undergo the procedures.
Sites / Locations
- John W. Sleasman