Status:
COMPLETED
Thymus Transplantation Safety-Efficacy
Lead Sponsor:
Sumitomo Pharma Switzerland GmbH
Collaborating Sponsors:
National Institutes of Health (NIH)
National Institute of Allergy and Infectious Diseases (NIAID)
Conditions:
Complete DiGeorge Anomaly
DiGeorge Syndrome
Eligibility:
All Genders
Phase:
NA
Brief Summary
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection. Withou...
Detailed Description
Complete DiGeorge anomaly (cDGA) is a congenital disorder characterized by athymia. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequen...
Eligibility Criteria
Inclusion
- Inclusion criteria for implantation of cultured thymus tissue:
- Must have 1 of following: 22q11.2ds or 10p13 hemizygosity; hypocalcemia requiring replacement; congenital heart disease; or CHARGE syndrome or CHD7 mutation
- Complete DiGeorge: \<50 CD3+ T cells/cumm or \<50 CD3+ T cells/cumm that are CD62L+ CD45RA+, or \<5% of CD3+ cells are CD62L+ CD45RA+
- Atypical DiGeorge subjects must have, or have had, a rash.
- Group 1
- •Typical cDGA whose T cells have a phytohemagglutinin (PHA) response of \< 5,000 counts per minute (cpm) and \< 20 fold PHA response.
- Group 2
- •Typical cDGA whose T cells have a PHA response of \>5,000 cpm and \<50,000 cpm and \>20 fold PHA response.
- Group 3
- Typical cDGA whose T cells have a PHA response of \>50,000 cpm.
- Typical cDGA with maternal engraftment
- Atypical cDGA whose T cells have a PHA response of \<40,000 cpm when on immunosuppression or \<75,000 cpm to PHA when not on immunosuppression.
- Atypical cDGA with group 3 PHA response \& maternal engraftment
- Group 4
- Atypical cDGA with PHA responses of \>75,000 cpm while on no immunosuppression or a PHA responses of \>40,000 cpm while on immunosuppression.
- Atypical cDGA with maternal engraftment and group 4 PHA response
- Exclusion criteria for implantation of cultured thymus tissue:
- Heart surgery conducted less than 4 weeks prior to projected implantation date.
- Heart surgery anticipated within 3 months after the proposed time of implantation
- Rejection by surgeon or anesthesiologist as surgical candidate
- Lack of sufficient muscle tissue to accept a transplant
- HIV infection
- Prior attempts at immune reconstitution, such as bone marrow transplant or previous thymus transplant
- CMV infection: For Groups 2, 3, and 4 CMV infection documented by \>500 copies/ml in the blood by PCR on two consecutive assays.
- Ventilator Dependence or Positive Pressure Support: Ventilator support or positive pressure support, such as Continuous Positive Airway Pressure (CPAP) or Bi-level Positive Airway Pressure (BiPAP) support for a condition that is deemed to be severe or irreversible or which renders the subject too clinically unstable to undergo the procedures.
Exclusion
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2023
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT01220531
Start Date
December 1 2010
End Date
August 1 2023
Last Update
January 16 2025
Active Locations (1)
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1
John W. Sleasman
Durham, North Carolina, United States, 27710