Complete DiGeorge Anomaly, Complete DiGeorge Syndrome, Congenital Athymia
Conditions
Keywords
Congenital Athymia
Brief 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).
Detailed 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 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.
Interventions
BIOLOGICALCultured Thymus Tissue
Product will be surgically administered into the quadriceps
Sponsors
Sumitomo Pharma Switzerland GmbH
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
0 Years to 21 Years
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.
Design outcomes
Primary
| Measure | 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
| Measure | Time frame |
|---|---|
| AESI classified by MedDRA coding, severity and grade | Immediately after the intervention/procedure/surgery |
Countries
United States
Contacts
Primary ContactJohn Sleasman, M.D.
Outcome results
None listed