Ignite Creation Date:
2025-12-24 @ 5:03 PM
Ignite Modification Date:
2026-02-21 @ 6:45 PM
Study NCT ID:
NCT05754450
Status:
RECRUITING
Last Update Posted:
2024-11-01
First Post:
2023-02-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects with Leukocyte Adhesion Deficiency Type II
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535887', 'term': 'Leukocyte adhesion deficiency type 1'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 2}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-04-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2024-12-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-10-30', 'studyFirstSubmitDate': '2023-02-06', 'studyFirstSubmitQcDate': '2023-03-02', 'lastUpdatePostDateStruct': {'date': '2024-11-01', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-03-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of treatment emergent adverse events reported during the study attributable to AVTX-803.', 'timeFrame': 'Through study completion, an average of 1 year.'}], 'secondaryOutcomes': [{'measure': 'Sialyl-Lewis X antigen expression on leukocytes', 'timeFrame': 'At 6 months'}, {'measure': 'Change from Baseline in the composite Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)', 'timeFrame': 'Through study completion, an average of 1 year.', 'description': 'The NPCRS comprises 3 sections, with each section containing specific items relevant to the assessment of subjects with CDG. Section I (Current Function); Section II (System Specific Involvement); and Section III (Current Clinical Assessment). The sum of the scores is used to scale patients into 3 domains: mild (0-14), moderate (15-25), and severe (\\>26) category.'}, {'measure': 'Change from Baseline in the individual parameters of the Nijmegen Pediatric Congenital Disorders of Glycosylation (CDG) Rating Scale (NPCRS)', 'timeFrame': 'Through study completion, an average of 1 year.', 'description': 'The NPCRS comprises 3 sections, with each section containing specific items relevant to the assessment of subjects with CDG. Section I (Current Function); Section II (System Specific Involvement); and Section III (Current Clinical Assessment). The sum of the scores is used to scale patients into 3 domains: mild (0-14), moderate (15-25), and severe (\\>26) category.'}, {'measure': 'Goal Attainment Score (GAS)', 'timeFrame': 'Through study completion, an average of 1 year.', 'description': 'The parent (or subject) formulates 3 individual goals for improving quality of life. Typically, these goals affect mobility, independence, and disease-related health aspects. These goals will be re-evaluated and scored by the parent or subject at each study visit within a scale of -2 to +2.'}, {'measure': 'Clinician Global Impression of Severity (CGI-S)', 'timeFrame': 'Through study completion, an average of 1 year.', 'description': 'The CGI-S scale is a 7-point scale that assesses the severity of the subject\'s severity of disease/illness. 1=Normal, not ill at all; 2=borderline ill; 3=mildly ill; 4=moderately ill; 5=markedly ill; 6= severely ill; 7=among the most extreme ill."'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Leukocyte Adhesion Deficiency']}, 'descriptionModule': {'briefSummary': 'The primary objective of this extension study is to assess the long-term safety and efficacy of AVTX-803 in subjects with LAD II (SLC35C1-CDG).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Subject must have completed protocol AVTX-803-LAD-301\n* Subject has biochemically and genetically proven LAD II (SLC35C1-CDG)\n\nExclusion Criteria:\n\n* Subject has severe anemia defined as hemoglobin \\<8.0 g/dL (\\<4.9 mmol/L)\n* Subject has impaired renal function as defined by an eGFR \\<90 mL/min\n* Subject has known or suspected intolerance or hypersensitivity to fucose or any ingredients of the investigational product\n* In the investigator's opinion, subject has a history of failure to respond to fucose at adequate dosing"}, 'identificationModule': {'nctId': 'NCT05754450', 'briefTitle': 'An Extension Study Assessing the Safety and Efficacy of AVTX-803 in Subjects with Leukocyte Adhesion Deficiency Type II', 'organization': {'class': 'INDUSTRY', 'fullName': 'AUG Therapeutics'}, 'officialTitle': 'A Phase 3, Open-Label, Extension Study to Assess the Long-term Safety and Efficacy of AVTX-803 in Subjects with Leukocyte Adhesion Deficiency Type II (LAD II)', 'orgStudyIdInfo': {'id': 'AVTX-803-LAD-302'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'AVTX-803', 'description': 'AVTX-803 at a dose specified by the investigator up to 5 times a day not to exceed 1700 mg/kg/day', 'interventionNames': ['Drug: AVTX-803']}], 'interventions': [{'name': 'AVTX-803', 'type': 'DRUG', 'description': 'L-fucose crystalline powder', 'armGroupLabels': ['AVTX-803']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Eva Morava-Kozicz, MD PhD', 'role': 'CONTACT'}], 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}], 'centralContacts': [{'name': 'Jennifer Lin', 'role': 'CONTACT', 'email': 'huiyil@Augtx.com', 'phone': '3052833827'}], 'overallOfficials': [{'name': 'David Deyle', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Mayo Clinic'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AUG Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}