Clinical trial NCT05329935: Congenital Athymia Patient Registry
Official title: Congenital Athymia Patient Registry of RETHYMIC
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Complete DiGeorge Anomaly
Complete DiGeorge Syndrome
Congenital Athymia
Keywords: Congenital Athymia
Lead sponsor: Sumitomo Pharma Switzerland GmbH
Sponsor class: INDUSTRY
First posted: 2022-04-15
Enrollment: 75
Sex: ALL
Minimum age (years): 0
Maximum age (years): 21
Standard ages: CHILD
ADULT
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
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.

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.

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).

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.

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.

Primary outcomes:
measure: Vital Status
timeFrame: 12 months post treatment with RETHYMIC.
measure: Flow cytometry including total and naïve CD3, CD4, and CD8 counts
timeFrame: Immediately after the intervention/procedure/surgery
Secondary outcomes:
measure: AESI classified by MedDRA coding, severity and grade
timeFrame: Immediately after the intervention/procedure/surgery
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke University School of Medicine
state: North Carolina
Study officials:
References:
citation: RETHYMIC US Prescribing Information, 2021.