Expanded Access Protocol Thymus Transplantation

• Status: Available
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

Details

• Lead Sponsor: M. Louise Markert
• Collaborator: Enzyvant Therapeutics GmbH
• Treatments: Antilymphocyte Serum; Cyclosporine; Cyclosporins; Tacrolimus; Thymoglobulin

Criteria

Cultured Thymus Tissue Inclusion Criteria for Implantation:

• an immunodeficiency or severe autoimmunity for which development of naïve T cells would be expected to lead to lead to clinical improvement.

• written consent (or consent of parent/legal guardian as applicable), review of medical testing, laboratory studies, and physical examinations are used to determine whether the patient is clinically stable and will potentially benefit from receiving cultured thymus tissue. Each participant is reviewed with the Data Safety and Monitoring Board (DSMB).

Cultured Thymus Tissue Exclusion Criteria for Implantation:

• Unrepaired cyanotic congenital heart disease

• Uncontrolled infections. "Uncontrolled" is defined as requiring a ventilator, dialysis, or vasopressor support or anticipated as requiring such support within 6 months.

• Pregnancy

• For females of child-bearing potential, a serum pregnancy test is done after consent, at the same time another blood draw is done if possible.

• Females of child-bearing potential must agree to contraceptive measures as indicated in the consent form.

• A second serum pregnancy test is done within 48 hours prior to administration of study interventions involving FDA pregnancy class D drugs, chemotherapy drugs, or other drugs or interventions known to pose risks to a potential fetus.

• HIV Positive

• History of malignancy

• CMV Infection

• For subjects receiving immunosuppression as part of the implantation protocol, CMV infection as documented by >500 copies/ml in blood by PCR on two consecutive assays is an exclusion.