Congenital Athymia Patient Registry
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-21
SponsorSumitomo Pharma Switzerland GmbH
About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
Written informed consent cannot be obtained.
Trial 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.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Cultured Thymus Tissue
Biological/VaccineProduct will be surgically administered into the quadriceps
Treatment groups
75 Participants
are divided into 2 treatment groupsGroup A: Main cohort1 intervention
Group B: Secondary cohort1 intervention
Trial outcomes
Primary outcomes
1
Vital Status
Time frame
12 months post treatment with RETHYMIC.2
Flow cytometry including total and naïve CD3, CD4, and CD8 counts
Time frame
Immediately after the intervention/procedure/surgerySecondary outcomes
1
AESI classified by MedDRA coding, severity and grade
Time frame
Immediately after the intervention/procedure/surgerySponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov