One purpose of this study is to determine whether the amount of cultured thymus tissue implanted into DiGeorge anomaly infants has any effect on the immune outcome. Another purpose of this study is to determine whether parental parathyroid transplantation (in addition to cultured thymus tissue implantation (CTTI) can help both the immune and the calcium problems in DiGeorge infants with hypocalcemia. \[Funding Source - FDA Office of Orphan Products Development (OOPD)\]
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 Transplant Inclusion Criteria:
* A parent or guardian of the DGS subject signed the consent form.
* Medical screening was completed.
* For a diagnosis of DGS, the subject had to have one of the following:
* Congenital heart disease;
* Hypocalcemia requiring replacement;
* 22q11.2 hemizygosity or 10p13 hemizygosity;
* CHARGE association or CHD7 mutation;
* A subject with abnormal ears whose mother had diabetes (type I, type II, or gestational).
* To meet the criteria of typical complete DiGeorge Anomaly (cDGA), the subject had to have either:
* Circulating CD3+ T cell count by flow cytometry \< 50/mm3 OR
* Circulating CD3+ T cells that were also positive for Cluster of Differentiation 45RA (CD45RA)+ CD62L+ and were \< 50/mm3 or less than 5% of total T cells.
Thymus Transplant Exclusion Criteria:
* Had heart surgery less than 4 weeks prior to projected implant date;
* Heart surgery anticipated within 3 months after the proposed time of implantation;
* Present or past lymphadenopathy;
* Rash associated with T cell infiltration of the dermis and epidermis;
* Rejection by the surgeon or anesthesiologist as surgical candidate;
* Lack of sufficient muscle tissue to accept a transplant of 4 g/m2 body surface area (BSA) or 0.2 g/kg subject bodyweight;
* Had human immunodeficiency virus (HIV) infection;
* Had prior attempts at immune reconstitution, such as bone marrow transplant or previous thymus transplantation;
* Ventilator support or positive pressure sup…
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.