Expanded Access Protocol Thymus Transplantation

Phase 4

Completed

• Conditions: Immunoreconstitution; Poor Thymic Function; Immunodeficiency; Athymia; Thymus Transplantation; Low T Cell Numbers
• Interventions: Biological: Cultured Thymus Tissue; Procedure: Blood Draw; Drug: Rabbit Anti-Thymocyte Globulin and Cyclosporine or Tacrolimus

• Registration Number: NCT02274662
• Lead Sponsor: Sumitomo Pharma Switzerland GmbH

Brief 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.

Detailed 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.

Study Timeline

• Study Start: 2014-08
• Study First Submitted: 2014-10-22
• Study First Submitted QC: 2014-10-23
• Study First Posted: 2014-10-24
• Primary Completion: 2023-08
• Study Completion: 2023-08
• Disposition First Submitted: 2024-07-29
• Disposition First Posted: 2024-07-29
• Status Verified: 2024-12
• Results First Submitted: 2024-12-19
• Results First Submitted QC: 2024-12-19
• Last Update Submitted: 2024-12-19
• Results First Posted: 2025-01-16
• Last Update Posted: 2025-01-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cultured thymus tissue implantation (CTTI)	Blood Draw	Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
Cultured thymus tissue implantation (CTTI)	Rabbit Anti-Thymocyte Globulin and Cyclosporine or Tacrolimus	Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
Cultured thymus tissue implantation (CTTI)	Cultured Thymus Tissue	Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders

Primary Outcome Measures

Name	Time	Method
Survival Rate at End of 1 Year	1 Year
Survival Rate at End of 2 Year	2 Year