NCT00579709

Thymus Transplantation With Immunosuppression

Thymus Transplantation With Immunosuppression, #884

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Sumitomo Pharma Switzerland GmbH · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed description

DiGeorge anomaly is a complex of cardiac defects, parathyroid deficiency, and thymus absence, resulting in profound T-cell deficiency. There is a spectrum of disease in DiGeorge anomaly with respect to all three defects. For complete DiGeorge anomaly subjects with severe T cell defect, the PI had shown that thymus transplantation is safe and efficacious without pretransplantation immunosuppression and with pretransplantation Thymoglobulin and cyclosporine. Some DiGeorge patients have very poor T cell function and are at risk of death from infection or other immune problems; however, these patients have enough T cell function to reject grafts. This protocol was designed for these patients. Atypical phenotype and some typical phenotype DiGeorge subjects were included in this protocol. Atypical complete DiGeorge anomaly patients have rash, lymphadenopathy, and oligoclonal T cell proliferations. The T cells have no markers of thymic function (they do not co-express CD45RA and CD62L; they do not contain T cell receptor rearrangement excision circles, TRECs). Typical complete DiGeorge anomaly patients in this protocol are those whose PHA response \>20 fold. Although these patients have very low T cell function, it may be enough to reject a transplant, so Thymoglobulin was used.

Conditions

• DiGeorge Syndrome
• DiGeorge Anomaly
• Complete DiGeorge Anomaly
• Complete DiGeorge Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	Thymus Tissue for Transplantation	3 Thymoglobulin doses given prior to thymus tx. Atypical subjects given Cyclosporine (Csa) pre-tx. Desired Csa concentration 180-300ng/ml. If post-tx T cell count remained \<4000/cumm Csa weaned over 8 weeks. If T cell \>4,000/cumm, Csa held at 180-300ng/ml. Thymus tissue, donor, \& mother of donor were screened for transplant safety. In operating room, thymic slices were transplanted into quadriceps muscle in 1 or both legs. Subjects had routine blood research immune evaluations. 2-3 months post-tx, open biopsy of allograft. Immune blood studies continued on surviving subjects until January 2010. Biological Mother: Mother provided blood sample used for DNA extraction, to identify/look for maternal T cell presence in recipient pre-tx, and/or for immune testing post-tx.

Timeline

• Start date: 2002-07-01
• Primary completion: 2006-12-01
• Completion: 2019-12-01
• First posted: 2007-12-24
• Last updated: 2022-04-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study