Parathyroid and Thymus Transplantation in DiGeorge #931 (NCT00566488) | Clinical Trial Compass
CompletedPhase 1
Parathyroid and Thymus Transplantation in DiGeorge #931
United States25 participantsStarted 2005-01
Plain-language summary
This study has three primary purposes: to assess parathyroid function after parathyroid transplantation in infants with Complete DiGeorge syndrome; to assess immune function development after transplantation; and, to assess safety and tolerability of the procedures. This is a Phase 1, single site, open, non-randomized clinical protocol. Enrollment is closed and study intervention is complete for all enrolled subjects; but subjects continue for observation and follow-up. Subjects under 2 years old with complete DiGeorge syndrome (atypical or typical) received thymus transplantation. Subjects received pre-transplant immune suppression with rabbit anti-human-thymocyte-globulin. Subjects with hypoparathyroidism and an eligible parental donor received thymus and parental parathyroid transplantation. A primary hypothesis: Thymus/Parathyroid transplant subjects will need less calcium and/or calcitriol supplementation at 1 year post-transplant as compared to historical controls.
Who can participate
Age range
24 Months
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.
Transplant Inclusion:
* Complete DiGeorge syndrome (typical or atypical) - may have DiGeorge as part of 22q11 hemizygosity, CHARGE association, or diabetic embryopathy or they may have no associated syndromes.
* Must have 1 of following:
* Circulating CD3+ T cells \< 50/mm3; or
* Circulating CD3+ T cells that also positive for CD45RA and CD62L must be \<50/mm3 or must be \< 5% of total T cells.
* Must be \<24 months old
* Laboratory studies must be done w/in 1 month of treatment:
* Thyroid studies - if abnormal must be on therapy, if recommended by endocrinology:
* PT and PTT must be \<2x upper limits of normal (ULN)
* Absolute neutrophil count must be \>500/mm3
* Platelet count must be \>50,000/mm3
* AST and ALT must be \<5x ULN
* Creatinine must be \<1.5 mg/dl
* Parents must agree to have infant stay in Durham until thymus biopsy is done 2-3 months post-treatment.
* Typical subjects must not have a rash with T cells on biopsy nor lymphadenopathy.
* Atypical subjects have rash with T cells on biopsy; may have lymphadenopathy.
* PHA proliferative responses must be tested 2x • Atypical: PHA response must be \<75,000cpm on 2 tests; test can be done while on immunosuppression.
Additional Criteria for Parathyroid Treatment Inclusion
* Hypoparathyroidism
* At least 1 parent must agree to be parathyroid donor
* Must require calcium supplementation to maintain ionized calcium \>1.0 mmol/L. Alternatively, intact PTH must be \<lower limit of normal when ionized ca…
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.
What they're measuring
1
Efficacy parameter: use of calcium/calcitriol at 1 year post-transplantation.