Clinical trial · Interventional
Expanded Access Protocol Thymus Transplantation
Expanded Access Protocol Thymus Transplantation for Immunodeficiency, Hematologic Malignancies, and Autoimmune Disease Related to Poor Thymic Function
NCT02274662CI-TRIAL-00084790completedPhase 4Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (6)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Athymia | — | UNRESOLVED | — |
| Immunodeficiency | — | UNRESOLVED | — |
| Immunoreconstitution | — | UNRESOLVED | — |
| Low T Cell Numbers | — | UNRESOLVED | — |
| Poor Thymic Function | — | UNRESOLVED | — |
| Thymus Transplantation | — | UNRESOLVED | — |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Blood Draw | Procedure | — | UNRESOLVED |
| Cultured Thymus Tissue | Biological | — | UNRESOLVED |
| Rabbit Anti-Thymocyte Globulin and Cyclosporine or Tacrolimus | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Cultured thymus tissue implantation (CTTI)
- description
- Cultured thymus tissue for the treatment of immunodeficiency and autoimmune disorders
- interventionNames
- Biological: Cultured Thymus Tissue
- Procedure: Blood Draw
- Drug: Rabbit Anti-Thymocyte Globulin and Cyclosporine or Tacrolimus
Primary outcomes (2)
- measure
- Survival Rate at End of 1 Year
- timeFrame
- 1 Year
- measure
- Survival Rate at End of 2 Year
- timeFrame
- 2 Year
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
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.
References
Publications (24)
- BACKGROUNDMarkert ML, Devlin BH, Alexieff MJ, Li J, McCarthy EA, Gupton SE, Chinn IK, Hale LP, Kepler TB, He M, Sarzotti M, Skinner MA, Rice HE, Hoehner JC. Review of 54 patients with complete DiGeorge anomaly enrolled in protocols for thymus transplantation: outcome of 44 consecutive transplants. Blood. 2007 May 15;109(10):4539-47. doi: 10.1182/blood-2006-10-048652. Epub 2007 Feb 6. PMID 17284531
- BACKGROUNDMarkert ML, Devlin BH, McCarthy EA. Thymus transplantation. Clin Immunol. 2010 May;135(2):236-46. doi: 10.1016/j.clim.2010.02.007. Epub 2010 Mar 16. PMID 20236866
- BACKGROUNDMarkert ML, Alexieff MJ, Li J, Sarzotti M, Ozaki DA, Devlin BH, Sedlak DA, Sempowski GD, Hale LP, Rice HE, Mahaffey SM, Skinner MA. Postnatal thymus transplantation with immunosuppression as treatment for DiGeorge syndrome. Blood. 2004 Oct 15;104(8):2574-81. doi: 10.1182/blood-2003-08-2984. Epub 2004 Apr 20. PMID 15100156
- BACKGROUNDSelim MA, Markert ML, Burchette JL, Herman CM, Turner JW. The cutaneous manifestations of atypical complete DiGeorge syndrome: a histopathologic and immunohistochemical study. J Cutan Pathol. 2008 Apr;35(4):380-5. doi: 10.1111/j.1600-0560.2007.00816.x. PMID 18333898
- BACKGROUNDChinn IK, Devlin BH, Li YJ, Markert ML. Long-term tolerance to allogeneic thymus transplants in complete DiGeorge anomaly. Clin Immunol. 2008 Mar;126(3):277-81. doi: 10.1016/j.clim.2007.11.009. Epub 2007 Dec 26. PMID 18155964
- BACKGROUNDMarkert ML, Li J, Devlin BH, Hoehner JC, Rice HE, Skinner MA, Li YJ, Hale LP. Use of allograft biopsies to assess thymopoiesis after thymus transplantation. J Immunol. 2008 May 1;180(9):6354-64. doi: 10.4049/jimmunol.180.9.6354. PMID 18424759
- BACKGROUNDHudson LL, Louise Markert M, Devlin BH, Haynes BF, Sempowski GD. Human T cell reconstitution in DiGeorge syndrome and HIV-1 infection. Semin Immunol. 2007 Oct;19(5):297-309. doi: 10.1016/j.smim.2007.10.002. Epub 2007 Nov 26.