Congenital Athymia Patient Registry

Congenital Athymia Patient Registry of RETHYMIC

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).

Study Overview

• Status: Recruiting
• Conditions: Complete DiGeorge Anomaly; Complete DiGeorge Syndrome; Congenital Athymia
• Intervention / Treatment: Biological: Cultured Thymus Tissue

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 Type: Observational
• Enrollment (Estimated): 75

Contacts and Locations

• Study Contact: Name: John Sleasman, M.D.; Phone Number: 919-684-9914; Email: john.sleasman@duke.edu

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Recruiting
      • Duke University School of Medicine
      • Contact: John Sleasman, M.D.; Phone Number: 919-684-9914; Email: john.sleasman@duke.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 21 years (Child, Adult)
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Main cohort; All eligible patients	Biological: Cultured Thymus Tissue; Product will be surgically administered into the quadriceps; Other Names: RETHYMIC; allogeneic cultured thymus tissue-agdc
Secondary cohort; All eligible patients who survived 1 year post treatment	Biological: Cultured Thymus Tissue; Product will be surgically administered into the quadriceps; Other Names: RETHYMIC; allogeneic cultured thymus tissue-agdc

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Time Frame
AESI classified by MedDRA coding, severity and grade	Immediately after the intervention/procedure/surgery

Collaborators and Investigators

• Sponsor: Sumitomo Pharma Switzerland GmbH

Publications and helpful links

General Publications

• RETHYMIC US Prescribing Information, 2021.

Study record dates

Study Major Dates

• Study Start (Actual): May 25, 2022
• Primary Completion (Estimated): April 1, 2026
• Study Completion (Estimated): April 1, 2026

Study Registration Dates

• First Submitted: March 24, 2022
• First Submitted That Met QC Criteria: April 8, 2022
• First Posted (Actual): April 15, 2022

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 13, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Congenital Athymia
• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Lymphatic Diseases; Endocrine System Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Parathyroid Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Craniofacial Abnormalities; Musculoskeletal Abnormalities; Abnormalities, Multiple; Chromosome Disorders; 22q11 Deletion Syndrome; Lymphatic Abnormalities; Hypoparathyroidism; DiGeorge Syndrome
• Other Study ID Numbers: RVT-802-4001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No