NCT02274662

Expanded Access Protocol Thymus Transplantation

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Sumitomo Pharma Switzerland GmbH · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

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

Interventions

Type	Name	Description
BIOLOGICAL	Cultured Thymus Tissue	Subjects receive cultured thymus tissue which is implanted into the quadriceps muscle. Subjects may receive pre and/or post-implantation immunosuppression. Potential subjects are screened for eligibility. The thymus tissue (from an unrelated donor), the donor, and the donor's mother are screened for safety. Cultured thymus tissue is implanted into the subject's quadriceps muscle under general anesthesia in the operating room. Two to three months post-implantation, if medically stable, subjects may undergo an allograft biopsy. Subjects undergo laboratory testing for approximately one year post-implantation. At year 2 post-implantation, subjects are contacted for data collection.
PROCEDURE	Blood Draw
DRUG	Rabbit Anti-Thymocyte Globulin and Cyclosporine or Tacrolimus	RATGAM and Cyclosporine or Tacrolimus may be given, depending on the patient. The doses, timing, and trough levels will vary depending on the patient's clinical condition.

Timeline

• Start date: 2014-08-01
• Primary completion: 2023-08-01
• Completion: 2023-08-01
• First posted: 2014-10-24
• Last updated: 2025-01-16
• Results posted: 2025-01-16