Congenital Athymia Patient Registry

Complete DiGeorge Syndrome Clinical Trial

Official title: Congenital Athymia Patient Registry of RETHYMIC

Status Recruiting

Clinical Trial 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).

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Complete DiGeorge Anomaly
• Complete DiGeorge Syndrome
• DiGeorge Syndrome

• NCT number: NCT05329935
• Study type: Observational
• Source: Sumitomo Pharma Switzerland GmbH
• Contact: John Sleasman, M.D.
• Phone: 919-684-9914
• Email: john.sleasman@duke.edu
• Status: Recruiting
• Start date: May 25, 2022
• Completion date: April 2026