Registry for patients with congenital athymia treated with RETHYMIC
Congenital Athymia Patient Registry of RETHYMIC
This study is tracking kids with congenital athymia who have received a new treatment called RETHYMIC to see how well it helps their immune system and overall health over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 75 (estimated) |
| Ages | 0 Years to 21 Years |
| Sex | All |
| Sponsor | Sumitomo Pharma Switzerland GmbH Industry-sponsored |
| Locations | 1 site (Durham, North Carolina) |
| Trial ID | NCT05329935 on ClinicalTrials.gov |
What this trial studies
This registry follows pediatric patients diagnosed with congenital athymia who have been treated with RETHYMIC, a cultured thymus tissue intervention. It employs a prospective cohort design to gather data on immune reconstitution, long-term survival, and adverse events following treatment. Patients are recruited within 60 days post-treatment or scheduled for treatment within 30 days. Clinical data and flow cytometry results will be collected at baseline and during follow-up at specified intervals.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients diagnosed with congenital athymia who are scheduled for or have recently undergone RETHYMIC treatment.
Not a fit: Patients who cannot provide informed consent or assent will not benefit from this study.
Why it matters
Potential benefit: If successful, this registry could provide valuable insights into the long-term effects of RETHYMIC treatment on immune function and survival in patients with congenital athymia.
How similar studies have performed: While this registry approach is observational, previous studies with RETHYMIC have shown promising results in immune reconstitution, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Durham, North Carolina
- Duke University School of Medicine — Durham, North Carolina, United States (Recruiting)
Study contacts
- Study coordinator: John Sleasman, M.D.
- Email: john.sleasman@duke.edu
- Phone: 919-684-9914
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.