Expanded Access Protocol Thymus Transplantation

Immunodeficiency Clinical Trial

Official title: Expanded Access Protocol Thymus Transplantation for Immunodeficiency, Hematologic Malignancies, and Autoimmune Disease Related to Poor Thymic Function

Status Approved for marketing

Clinical Trial Summary

The primary purpose is to provide access for patients who have immunodeficiency or severe autoimmune disease related to poor thymic function to cultured thymus tissue for implantation. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection.

Eligible participants receive cultured thymus tissue for implantation and may undergo biopsy. Immune suppression may be given depending on the immune status and clinical condition of the participant. Immune function testing is continued for one year post-implantation.

Clinical Trial Description

The patients enrolled have a high likelihood of death if they do not receive culture thymus tissue because of lack of thymus function. As there are many types of patients who may be enrolled, study results will not have statistical significance.

The study objective is to make cultured thymus tissue available for implantation on an expanded access basis. Data will be collected on survival, naïve T cell development, T cell chimerism, and implant related toxicities, as well as any unexpected study-related serious adverse events.

Eligible subjects receive cultured thymus tissue and may undergo allograft biopsy. Immune suppression may be given depending on the subject's immune status and clinical condition.

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

Related Conditions & MeSH terms

• Athymia
• Immunodeficiency
• Immunologic Deficiency Syndromes
• Immunoreconstitution
• Low T Cell Numbers
• Poor Thymic Function
• Thymus Transplantation

• NCT number: NCT02274662
• Study type: Expanded Access
• Source: Enzyvant Therapeutics GmBH
• Status: Approved for marketing