Congenital Athymia Patient Registry

Recruiting

• Conditions: Congenital Athymia; Complete DiGeorge Syndrome; Complete DiGeorge Anomaly
• Interventions: Biological: Cultured Thymus Tissue

• Registration Number: NCT05329935
• Lead Sponsor: Sumitomo Pharma Switzerland GmbH

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.

Study Timeline

• Study First Submitted: 2022-03-24
• Study First Submitted QC: 2022-04-08
• Study First Posted: 2022-04-15
• Study Start: 2022-05-25
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-13
• Last Update Posted: 2024-03-15
• Primary Completion: 2026-04
• Study Completion: 2026-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 75

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Main cohort	Cultured Thymus Tissue	All eligible patients
Secondary cohort	Cultured Thymus Tissue	All eligible patients who survived 1 year post treatment

Primary Outcome Measures

Name	Time	Method
Flow cytometry including total and naïve CD3, CD4, and CD8 counts	Immediately after the intervention/procedure/surgery
Vital Status	12 months post treatment with RETHYMIC.

Secondary Outcome Measures

Name	Time	Method
AESI classified by MedDRA coding, severity and grade	Immediately after the intervention/procedure/surgery

Trial Locations

Locations (1)

Duke University School of Medicine; 🇺🇸 Durham, North Carolina, United States