Congenital Athymia Patient Registry
1 other identifier
observational
75
1 country
1
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started May 2022
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 24, 2022
CompletedFirst Posted
Study publicly available on registry
April 15, 2022
CompletedStudy Start
First participant enrolled
May 25, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2026
CompletedAugust 19, 2025
August 1, 2025
3.9 years
March 24, 2022
August 14, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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 Outcomes (1)
AESI classified by MedDRA coding, severity and grade
Immediately after the intervention/procedure/surgery
Study Arms (2)
Main cohort
All eligible patients
Secondary cohort
All eligible patients who survived 1 year post treatment
Interventions
Product will be surgically administered into the quadriceps
Eligibility 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.
You may qualify if:
- 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.
You may not qualify if:
- Written informed consent cannot be obtained.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Duke University School of Medicine
Durham, North Carolina, 27710, United States
Related Publications (1)
RETHYMIC US Prescribing Information, 2021.
BACKGROUND
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- OTHER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 24, 2022
First Posted
April 15, 2022
Study Start
May 25, 2022
Primary Completion
April 1, 2026
Study Completion
April 1, 2026
Last Updated
August 19, 2025
Record last verified: 2025-08