Recruiting

Observational Study

Sponsor:

Sumitomo Pharma Switzerland GmbH

Code:

NCT05329935

Conditions

Complete DiGeorge Anomaly

Complete DiGeorge Syndrome

Congenital Athymia

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

Interventions

Cultured Thymus Tissue

Study Details

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).

Conditions

Complete DiGeorge Anomaly

Complete DiGeorge Syndrome

Congenital Athymia

Study ID

NCT05329935

Start date

May 25, 2022

Status verified date

Aug, 2025

Completion date

Apr, 2026

Anticipated

Primary completion date

Apr, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

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

Enrollment

75 participants

Anticipated

Interventions and Outcome Measures

Arms

Main cohort

All eligible patients

Secondary cohort

All eligible patients who survived 1 year post treatment

Interventions

Cultured Thymus Tissue

Product will be surgically administered into the quadriceps

Primary outcome measure

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

Central Contacts and Locations

Central contacts

John Sleasman, M.D.

919-684-9914john.sleasman@duke.edu

Locations

Duke University School of Medicine

Recruiting

Durham, North Carolina, United States, 27710

Contacts

John Sleasman, M.D.

919-684-9914john.sleasman@duke.edu

More Information

Sponsor

Sumitomo Pharma Switzerland GmbH

Last update posted

Aug 19, 2025

Last verified

Aug, 2025

Keywords

  • Congenital Athymia

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Sumitomo Pharma Switzerland GmbH on 2025-08-19.