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
Sex
ALL
See this in plain English?
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.