Ignite Creation Date:
2025-12-24 @ 10:22 PM
Ignite Modification Date:
2026-01-03 @ 10:42 AM
Study NCT ID:
NCT05329935
Status:
RECRUITING
Last Update Posted:
2025-08-19
First Post:
2022-03-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Congenital Athymia Patient Registry
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 75}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-05-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2026-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-14', 'studyFirstSubmitDate': '2022-03-24', 'studyFirstSubmitQcDate': '2022-04-08', 'lastUpdatePostDateStruct': {'date': '2025-08-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-04-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'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'}], 'secondaryOutcomes': [{'measure': 'AESI classified by MedDRA coding, severity and grade', 'timeFrame': 'Immediately after the intervention/procedure/surgery'}]}, 'oversightModule': {'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Congenital Athymia'], 'conditions': ['Complete DiGeorge Anomaly', 'Complete DiGeorge Syndrome', 'Congenital Athymia']}, 'referencesModule': {'references': [{'type': 'BACKGROUND', 'citation': 'RETHYMIC US Prescribing Information, 2021.'}]}, 'descriptionModule': {'briefSummary': '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.\n\nClinical 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).', 'detailedDescription': '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.\n\nClinical 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).\n\nCurrently, 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '0 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Pediatric patients diagnosed with Congenital Athymia:\n\n• Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.', 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Pediatric patients diagnosed with Congenital Athymia:\n\n * Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.\n * Who have provided written informed consent directly, or written informed consent has been provided by the patient, the parent, or legal guardian.\n\nExclusion Criteria:\n\n* Written informed consent cannot be obtained.'}, 'identificationModule': {'nctId': 'NCT05329935', 'briefTitle': 'Congenital Athymia Patient Registry', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sumitomo Pharma Switzerland GmbH'}, 'officialTitle': 'Congenital Athymia Patient Registry of RETHYMIC', 'orgStudyIdInfo': {'id': 'RVT-802-4001'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Main cohort', 'description': 'All eligible patients', 'interventionNames': ['Biological: Cultured Thymus Tissue']}, {'label': 'Secondary cohort', 'description': 'All eligible patients who survived 1 year post treatment', 'interventionNames': ['Biological: Cultured Thymus Tissue']}], 'interventions': [{'name': 'Cultured Thymus Tissue', 'type': 'BIOLOGICAL', 'otherNames': ['RETHYMIC', 'allogeneic cultured thymus tissue-agdc'], 'description': 'Product will be surgically administered into the quadriceps', 'armGroupLabels': ['Main cohort', 'Secondary cohort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'John Sleasman, M.D.', 'role': 'CONTACT', 'email': 'john.sleasman@duke.edu', 'phone': '919-684-9914'}], 'facility': 'Duke University School of Medicine', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}], 'centralContacts': [{'name': 'John Sleasman, M.D.', 'role': 'CONTACT', 'email': 'john.sleasman@duke.edu', 'phone': '919-684-9914'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sumitomo Pharma Switzerland GmbH', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}