Congenital Athymia Patient Registry | NCT05329935

Studies
Congenital Athymia Patient Registry

NCT05329935

Sponsor

Sumitomo Pharma Switzerland GmbH

Status

Recruiting

Last Update Posted

Aug 19, 2025Actual

Enrollment

75Estimated

Phase

Not provided

Conditions

Complete DiGeorge Anomaly
Complete DiGeorge Syndrome
Congenital Athymia

Interventions

Cultured Thymus Tissue

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 25, 2022Actual

Primary Completion Date

Apr 2026Estimated

Completion Date

Apr 2026Estimated

First Submitted Date

Mar 24, 2022

First Submission Date that Met QC Criteria

Apr 8, 2022

First Posted Date

Apr 15, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 14, 2025

Last Update Posted Date

Aug 19, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

75Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Main cohort		All eligible patients	Biological: Cultured Thymus Tissue
Secondary cohort		All eligible patients who survived 1 year post treatment	Biological: Cultured Thymus Tissue

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Cultured Thymus Tissue	Biological	Product will be surgically administered into the quadriceps	Main cohort Secondary cohort

Outcomes Module

Primary Outcomes

Measure	Description	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 Outcomes

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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

0 Years

Maximum Age

21 Years

Standard Ages

ChildAdult

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.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
John Sleasman, M.D.	Contact	919-684-9914		john.sleasman@duke.edu

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Duke University School of Medicine	Recruiting	Durham	North Carolina	27710	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
	Background	RETHYMIC US Prescribing Information, 2021.