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Clinical Trials/NCT00579709
NCT00579709CompletedPhase 1

Thymus Transplantation With Immunosuppression, #884

Sumitomo Pharma Switzerland GmbH2 sites in 1 country15 target enrollmentStarted: July 2002Last updated:
Conditions

Trial Snapshot

Phase
Phase 1
Status
Completed
Enrollment
15
Locations
2
Primary Endpoint
Safety & tolerability of Thymoglobulin and cyclosporine followed by thymus transplantation: Survival at 1 year post-transplantation.

Study Overview

Brief 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.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Not provided

Exclusion Criteria

  • Not provided

Outcomes

Primary Outcomes

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

Time Frame: 1 year post-transplantation

Secondary Outcomes

  • Allograft biopsy used to evaluate graft rejection(2 to 4 months post-transplant)
  • Use of additional post transplant immunosuppression after that listed in the protocol.(The post thymus transplantation period)
  • CD3 count(10 - 14 months post-transplantation)
  • CD8 count(10-14 months after thymus transplantation)
  • Thymopoiesis(2-4 months after thymus transplantation)
  • CD4 count(10-14 months after thymus transplantation)
  • naive CD4 count(10-14 months after thymus transplantation)
  • naive CD8 count(10-14 months after thymus transplantation)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (2)

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