Observational Registry Study of RETHYMIC Treatment for Children and Young Adults with Congenital Athymia

What this Trial Is About

Researchers are observing patients with Congenital Athymia who have been treated with RETHYMIC in this registry study. The goal is to understand the immune recovery process following treatment, the long-term survival of patients, and any special adverse events that may occur. RETHYMIC has shown potential to help rebuild the immune system and extend life in previous clinical studies. Patients receive an investigational treatment called Cultured Thymus Tissue, which is surgically implanted into the quadriceps muscle. The study follows patients prospectively, including those recently treated or scheduled to receive treatment within specific time frames. The registry collects medical history and clinical data from patient records at baseline and during follow-up visits. Participants are monitored starting the day after their treatment surgery. Researchers review clinical data and flow cytometry tests that measure immune cell counts. The main outcomes include vital status 12 months after treatment and detailed immune cell measurements right after surgery. The study also tracks any adverse events classified by severity. Follow-up and data collection continue to better understand treatment impact over time.

Congenital Athymia Patient Registry