Congenital Athymia Patient Registry of RETHYMIC
Sumitomo Pharma Switzerland GmbH
Summary
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).
Description
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…
Eligibility
- Age range
- 0–21 years
- Sex
- All
- Healthy volunteers
- Not specified
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.
Interventions
- BiologicalCultured Thymus Tissue
Product will be surgically administered into the quadriceps
Location
- Duke University School of MedicineDurham, North Carolina