Phase I/II Thymus Transplantation With Immunosuppression #950
United States14 participantsStarted 2005-12-19
Plain-language summary
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and effective treatment for complete DiGeorge anomaly. This study will also evaluate whether cultured thymus tissue implantation and parathyroid transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly and hypoparathyroidism.
Who can participate
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Thymus Transplantation Inclusion:
* Must have 1 of following: 22q11 or 10p13 hemizygosity; hypocalcemia requiring replacement; congenital heart defect; CHARGE association or CHD7 mutation; or abnormal ears plus mother w/diabetes (type I, type II, gestational).
* \<50 CD3+ T cells/cumm or \<50 CD3+ T cells/cumm that are CD62L+ CD45RA+ (cluster of differentiation 45RA) (naïve phenotype), or \<5% of CD3+ count being CD62L+ CD45RA+
Atypical DiGeorge:
* Must have, or have had, a rash. If rash present, rash biopsy must show T cells in skin. If rash \& adenopathy resolved, must have \>50/cumm T cells \& naive T cell must be \<50/cumm or \<5% of T cells.
Typical DiGeorge:
* CD3+ CD45RA+ CD62L+ T cells \<50/mm3 or \<5% of total T cells
Parathyroid Transplantation Additional Inclusion:
* 2 studies in recipient which PTH\<5 pg/ml when ionized calcium \<1.1 mmol/L. Can be done anytime pre-tx; 1 must be done while at Duke Hospital.
* Parent(s) willing \& eligible to be donors
Thymus Transplantation Exclusion:
* Heart surgery \<4 wks pre-tx
* Heart surgery anticipated w/in 3 months after proposed tx
* Rejection by surgeon or anesthesiologist as surgical candidate
* Lack of sufficient muscle tissue to accept transplant of 4 grams/m2 BSA
* HIV infection
* Prior attempts at immune reconstitution, such as bone marrow tx or previous thymus tx
* CMV(\>500 copies/ml blood by PCR on 2 tests)
* Ventilator dependence
Parathyroid Donor Inclusion:
* \>18 years of age
* Serum calcium in norm…