Congenital Athymia Patient Registry

Conditions

• Complete DiGeorge Anomaly
• Complete DiGeorge Syndrome
• Congenital Athymia

Eligibility Criteria

Sex

ALL

Age

0 Years - 21 Years

Healthy Volunteers

Not accepted

Interventions

• Cultured Thymus Tissue — BIOLOGICAL
  Product will be surgically administered into the quadriceps

Study Details

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).

Key Dates

Start date

May 25, 2022

Status verified

Aug 2025

Primary completion

Apr 30, 2026

Completion

Apr 30, 2026

Study Design

Enrollment

75 participants (estimated)

Arms

• Arm: Main cohort
  All eligible patients
• Arm: Secondary cohort
  All eligible patients who survived 1 year post treatment

Primary Outcome Measure

Vital Status [ Time Frame: 12 months post treatment with RETHYMIC. ]

Central Contacts

• John Sleasman, M.D.
  919-684-9914
  [email protected]

Locations (1)

Find similar trials in Durham, NC