CompletedPhase 1

Thymus Transplantation With Immunosuppression

United States15 participantsStarted 2002-07

Plain-language summary

The research purpose is to determine if thymus transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly. The research includes studies to evaluate whether thymus transplantation results in complete DiGeorge anomaly subjects developing a normal immune system.

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.

Transplant Inclusion * No age limit * Thyroid studies must be done and if abnormal, must be on therapy DiGeorge diagnosis - must have 1 symptom from the following list: * Heart defect * Hypocalcemia requiring replacement * 22q11 hemizygosity * 10p13 hemizygosity * CHARGE association * Abnormal ears plus mother with diabetes (type I, type II, or gestational) Atypical Diagnosis: * Must have, or have had, a rash. If rash present, biopsy of rash must show T cells in skin. If rash \& adenopathy resolved, must still have oligoclonal T cells. * Within 1 month of tx must have PHA response \>20 fold above background or \>5,000 cpm, whichever is higher, or response can be \< this. * Circulating CD3+ T cells \>50/mm3 but CD45RA+CD62L+CD3+ T cells \<50/mm or \<5% of CD3 count, whichever is higher (must be done 2x) * Immunoscope with \>40% oligoclonal TCRBV families. A 2nd test per sponsor discretion if T cell numbers increase or activation status changes. * If TREC done pre-tx must have TRECs \<100 per 100,000 CD3+ cells. Typical Diagnosis: * Circulating CD3+ CD45RA+ CD62L+ T cells and \<50/mm3 or \<5% of total T cells * PHA response \>20 fold above background or \>5,000 cpm, whichever is higher. * 2 studies must show similar immunological findings qualify for this study. * TRECs, if done, should be \<100/100,000 CD3 cells Transplant Exclusion: * Heart surgery \<4 weeks pre-tx date * Heart surgery anticipated w/in 3 months of proposed tx * Rejection by surgeon or anesthesiologis…

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

Safety & tolerability of Thymoglobulin and cyclosporine followed by thymus transplantation: Survival at 1 year post-transplantation.

Timeframe: 1 year post-transplantation

Trial details

NCT IDNCT00579709

SponsorSumitomo Pharma Switzerland GmbH

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2006-12

View on ClinicalTrials.gov More DiGeorge Syndrome trials

Plain-language summary

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.