Thymus Transplantation Safety-Efficacy

DiGeorge Syndrome Clinical Trial

Official title: Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836

Status Approved for marketing

Clinical Trial Summary

Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequent death.

Cultured thymus tissue with and without immunosuppression (drugs given before and after implantation) has resulted in the development of good T cell function in subjects with complete DiGeorge anomaly.

This expanded access study continues cultured thymus tissue safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants receive cultured thymus tissue. Immune function testing is continued for one year post-implantation.

Clinical Trial Description

Complete DiGeorge anomaly (cDGA) is a congenital disorder characterized by athymia. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequent death. In patients with cDGA, implantation of cultured thymus tissue with and without immunosuppression has resulted in diverse T cell development and good T cell function.

The purpose of this expanded access study is to continue cultured thymus tissue safety and efficacy research for the treatment of athymia in patients with cDGA. Until administration of cultured thymus tissue is FDA approved as standard care for cDGA, research study participation is the only means by which a patient may have access to this potentially life-saving procedure.

This protocol includes 4 groups: one for subjects who do not require immunosuppression; and 3 immunosuppression groups for subjects with different T cell function levels to be suppressed adequately.

Eligible subjects receive cultured thymus tissue and may undergo an allograft biopsy.

Protocol specified studies continue until approximately one year post-implantation. Study participation lasts two years. ;

Related Conditions & MeSH terms

• Complete DiGeorge Anomaly
• Complete DiGeorge Syndrome
• Congenital Abnormalities
• Craniosynostoses
• DiGeorge Anomaly
• DiGeorge Syndrome
• Marfan Syndrome
• Syndrome

• NCT number: NCT01220531
• Study type: Expanded Access
• Source: Enzyvant Therapeutics GmBH
• Status: Approved for marketing