Expired Study
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Durham, North Carolina 27710


Purpose:

The study purpose is to determine whether thymus transplantation without immunosuppression is effective in treating typical complete DiGeorge syndrome.


Study summary:

There is no safe and effective treatment for DiGeorge syndrome and most patients die by the age of two. Complete DiGeorge syndrome is characterized by very low T cell or very low naïve T cell numbers. In this study, typical complete DiGeorge syndrome subjects received human postnatal cultured thymus tissue transplants. Thymus tissue that would otherwise be discarded was transplanted into DiGeorge subjects in the operating room. At the time of transplantation, a skin biopsy was obtained to look for any preexisting T cells. After transplantation, subjects were followed by routine research immune evaluations, using blood samples obtained every 2-4 weeks. At approximately 2-3 months post-transplantation subjects underwent an open biopsy of the allograft. The biopsy was done under general anesthesia in the operating room. At the time of the graft biopsy, another skin biopsy was obtained to look for clonal populations of T cells. The protocol aims include: assessing thymopoiesis in the allograft biopsy; assessing immunoreconstitution of complete DiGeorge syndrome subjects after postnatal allogeneic thymus transplantation; assessing minimally invasive methods of assessing thymopoiesis (flow cytometry and polymerase chain reaction (PCR); assessing pre-transplant T cells which do not proliferate in response to mitogens (focusing on NK-T cells); and, assessing thymus transplantation safety and toxicity.


Criteria:

Inclusion Criteria: - Diagnosis of complete DiGeorge syndrome which is either: T cells with < 50/mm3 with naive phenotype; or < 5% CD3 + T cells with naive T cell phenotype. - Diagnosis of typical DiGeorge syndrome phenotype: < 50 T cells/cumm and very low proliferative responses to mitogens (e.g. < 20 fold response to mitogen phytohemagglutinin). - Proliferative response to PHA < 20 fold above background or < 5000 counts per minute(cpm), whichever is higher. {Note: Subjects with PHA responses 20 fold or more over background or > 5,000 cpm, whichever is higher, may be enrolled in another thymus transplant protocol.} - Must have heart disease; hypocalcemia requiring replacement; 22q11 or 10p13 hemizygosity; CHARGE association; or must be child of diabetic mother and have abnormal ears. Exclusion Criteria: - Those who do not meet inclusion criteria - Atypical DiGeorge syndrome phenotype - Rash indicating atypical DiGeorge syndrome phenotype. Transplant Exclusion: - Heart surgery <4 weeks pre-tx date - Heart surgery anticipated w/in 3 months of proposed tx - Rejection by surgeon or anesthesiologist as surgical candidates - Lack of sufficient muscle tissue to accept 0.2 grams/kg transplant


NCT ID:

NCT00576407


Primary Contact:

Principal Investigator
M. Louise Markert, MD, PhD
Duke University Medical Center, Pediatrics, Allergy & Immunology


Backup Contact:

N/A


Location Contact:

Durham, North Carolina 27710
United States



There is no listed contact information for this specific location.

Site Status: N/A


Data Source: ClinicalTrials.gov

Date Processed: November 17, 2017

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