Expanded Access Protocol Thymus Transplantation

December 19, 2024 updated by: Sumitomo Pharma Switzerland GmbH

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

11

Phase

  • Phase 4

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cultured thymus tissue implantation (CTTI)
Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
Procedure: Blood Draw
Other Names:
  • Venipuncture
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.

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.
Other Names:
  • RATGAM and Csa or FK506

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Survival Rate at End of 1 Year
Time Frame: 1 Year
1 Year
Survival Rate at End of 2 Year
Time Frame: 2 Year
2 Year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sumitomo Pharma Switzerland GmbH

Investigators

  • Principal Investigator: John W. Sleasman, M.D., Duke University Medical Center, Pediatrics, Allergy & Immunology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2014

Primary Completion (Actual)

August 1, 2023

Study Completion (Actual)

August 1, 2023

Study Registration Dates

First Submitted

October 22, 2014

First Submitted That Met QC Criteria

October 23, 2014

First Posted (Estimated)

October 24, 2014

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

December 19, 2024

Last Verified

December 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Pro00051692

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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