Congenital Athymia Patient Registry

Complete DiGeorge Syndrome Clinical Trial

Official title:

Congenital Athymia Patient Registry of RETHYMIC

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05329935
• Other study ID #: RVT-802-4001
• Status: Recruiting
• Start date: May 25, 2022
• Est. completion date: April 2026

Study information

• Verified date: March 2024
• Source: Sumitomo Pharma Switzerland GmbH
• Contact: John Sleasman, M.D.
• Phone: 919-684-9914
• Email: john.sleasman[@]duke.edu
• Is FDA regulated: No
• Study type: Observational

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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 75
• Est. completion date: April 2026
• Est. primary completion date: April 2026
• Gender: All
• Age group: 0 Years to 21 Years

Eligibility

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.

Related Conditions & MeSH terms

• Complete DiGeorge Anomaly
• Complete DiGeorge Syndrome
• DiGeorge Syndrome

Intervention

Biological:
• Cultured Thymus Tissue
Product will be surgically administered into the quadriceps

Locations

Country	Name	City	State
United States	Duke University School of Medicine	Durham	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Sumitomo Pharma Switzerland GmbH

Country where clinical trial is conducted

United States, 

References & Publications (1)

RETHYMIC US Prescribing Information, 2021.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Vital Status		12 months post treatment with RETHYMIC.
Primary	Flow cytometry including total and naïve CD3, CD4, and CD8 counts		Immediately after the intervention/procedure/surgery
Secondary	AESI classified by MedDRA coding, severity and grade		Immediately after the intervention/procedure/surgery