Congenital Athymia Patient Registry

Study Overview
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Brief Summary
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This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC. Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).

Detailed Description
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This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC. Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI). Currently, there is a single site where patients are being treated with RETHYMIC. The Investigator will be responsible for recruiting patients who have recently had (i.e., within the past 60 days) or are scheduled to have this treatment within 30 days and obtaining signed informed consent (and assent as applicable). The Investigator will collect baseline medical history and clinical data information for each enrolled subject. The start of follow-up (day 1) begins on the day following treatment surgery. Clinical data and flow cytometry test results (when conducted) will be abstracted from the medical records by Clinical Staff at baseline and during follow-up at predefined intervals.

Official Title
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Congenital Athymia Patient Registry of RETHYMIC

Conditions
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Complete DiGeorge Anomaly, Complete DiGeorge Syndrome, Congenital Athymia

Intervention / Treatment
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* Biological: Cultured Thymus Tissue


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Pediatric patients diagnosed with Congenital Athymia: Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days. Who have provided written informed consent directly, or written informed consent has been provided by the patient, the parent, or legal guardian. Exclusion Criteria: Written informed consent cannot be obtained.

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Main cohort<br>All eligible patients | Biological: Cultured Thymus Tissue<br>* Product will be surgically administered into the quadriceps<br>* Other names: allogeneic cultured thymus tissue-agdc;|
| Secondary cohort<br>All eligible patients who survived 1 year post treatment | Biological: Cultured Thymus Tissue<br>* Product will be surgically administered into the quadriceps<br>* Other names: allogeneic cultured thymus tissue-agdc;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Vital Status |  | 12 months post treatment with RETHYMIC. | 
| Flow cytometry including total and naïve CD3, CD4, and CD8 counts |  | Immediately after the intervention/procedure/surgery | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AESI classified by MedDRA coding, severity and grade |  | Immediately after the intervention/procedure/surgery | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Congenital Athymia