N/A
N=29
Thymus Transplantation Safety-Efficacy
Complete DiGeorge Anomaly · DiGeorge Syndrome · DiGeorge Anomaly · Complete DiGeorge Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01220531 ↗Enrolled (actual)
29
Serious AEs
82.8%
Results posted
Jan 2025
Primary outcome: Primary: Survival Rate at End of 1 Year — 79.3 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Cultured Thymus Tissue (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Sumitomo Pharma Switzerland GmbH
- Primary completion
- Aug 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Survival Rate at End of 1 Year |
79.3 | — |
| PRIMARY Survival Rate at End of 2 Year |
75 | — |
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. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequent death.
Cultured thymus tissue with and without immunosuppression (drugs given before and after implantation) has resulted in the development of good T cell function in subjects with complete DiGeorge anomaly.
This expanded access study continues cultured thymus tissue safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants receive cultured thymus tissue. Immune function testing is continued for one year post-implantation.
Eligibility Criteria
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: 5,000 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 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.
Data sourced from ClinicalTrials.gov (NCT01220531). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.