Ignite Creation Date:
2025-12-25 @ 3:46 AM
Ignite Modification Date:
2025-12-26 @ 2:32 AM
Study NCT ID:
NCT05101902
Status:
COMPLETED
Last Update Posted:
2024-09-19
First Post:
2021-10-20
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000312', 'term': 'Adrenal Hyperplasia, Congenital'}], 'ancestors': [{'id': 'D047808', 'term': 'Adrenogenital Syndrome'}, {'id': 'D012734', 'term': 'Disorders of Sex Development'}, {'id': 'D014564', 'term': 'Urogenital Abnormalities'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D043202', 'term': 'Steroid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D006058', 'term': 'Gonadal Disorders'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 65}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-07-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-09', 'completionDateStruct': {'date': '2024-08-07', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-09-12', 'studyFirstSubmitDate': '2021-10-20', 'studyFirstSubmitQcDate': '2021-10-20', 'lastUpdatePostDateStruct': {'date': '2024-09-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-11-01', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-08-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants Potentially Eligible for CAH-301 Study', 'timeFrame': 'Up to 5 years'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['CAH'], 'conditions': ['Congenital Adrenal Hyperplasia']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://adrenastx.com', 'label': 'Description: Adrenas Therapeutics Website'}, {'url': 'https://cahgenetherapy.com', 'label': 'CAH Gene Therapy Website'}]}, 'descriptionModule': {'briefSummary': 'This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 \\[NCT04783181\\] gene therapy trial with BBP-631.', 'detailedDescription': 'Adult participants diagnosed with classic CAH due to 21-OHD and who are taking glucocorticoids as treatment for CAH will undergo assessments to determine their potential eligibility for participation in the adult CAH-301 treatment trial with BBP-631.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Adult participants diagnosed with classic CAH due to 21-OHD', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult male and non-pregnant females with classic CAH (simple virilizing or salt-wasting) due to 21-OHD\n* Screening/baseline 17-OHP levels \\> 5-10 × ULN\n* Is on a daily regimen of glucocorticoid\n* Naïve to prior gene therapy or AAV-mediated therapy\n\nExclusion Criteria:\n\n* Positive for anti-AAV5 antibodies\n* History of adrenalectomy and has no significant liver disease'}, 'identificationModule': {'nctId': 'NCT05101902', 'briefTitle': 'A Study to Determine Eligibility for CAH-301 (A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH) [NCT04783181])', 'organization': {'class': 'INDUSTRY', 'fullName': 'Adrenas Therapeutics Inc'}, 'officialTitle': 'Pre-screening Study to Identify Adult Participants with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial with BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene', 'orgStudyIdInfo': {'id': 'CAH-300'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Overall Study', 'description': 'Participants will undergo antibody and genetic screen, initial pre-screening, eligibility monitoring and eligibility confirmation for potential transition to the treatment trial center once a treatment assignment in the study CAH-301 becomes available. Participation in Study CAH-300 is permitted to continue until treatment assignment is available in the treatment trial or unless, during eligibility monitoring or eligibility', 'interventionNames': ['Other: Pre-Screening']}], 'interventions': [{'name': 'Pre-Screening', 'type': 'OTHER', 'description': 'No intervention given', 'armGroupLabels': ['Overall Study']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90094', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Science37', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Adrenas Therapeutics Inc', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}