Ignite Creation Date:
2025-12-25 @ 2:46 AM
Ignite Modification Date:
2026-03-05 @ 7:58 PM
Study NCT ID:
NCT03354533
Status:
COMPLETED
Last Update Posted:
2019-01-25
First Post:
2017-11-21
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535755', 'term': 'Congenital disorder of glycosylation, type 2C'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D005643', 'term': 'Fucose'}], 'ancestors': [{'id': 'D003837', 'term': 'Deoxy Sugars'}, {'id': 'D002241', 'term': 'Carbohydrates'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 4}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-01-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-01', 'completionDateStruct': {'date': '2018-11-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-01-22', 'studyFirstSubmitDate': '2017-11-21', 'studyFirstSubmitQcDate': '2017-11-24', 'lastUpdatePostDateStruct': {'date': '2019-01-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2017-11-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-11-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Decrease in infection frequency', 'timeFrame': '12 months after treatment started', 'description': 'Statistically significant decrease in infection frequency'}], 'secondaryOutcomes': [{'measure': 'Decrease in neutrophil count', 'timeFrame': '30 days after treatment started', 'description': 'Statistically significant decrease in absolute neutrophil count'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False}, 'conditionsModule': {'conditions': ['Leukocyte Adhesion Deficiency, Type II']}, 'descriptionModule': {'briefSummary': 'Study of ORL-1F in Patients With Leukocyte Adhesion Deficiency Type II'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of Leukocyte Adhesion Deficiency Type II.\n* Less than 18 years old.\n\nExclusion Criteria:\n\n* Diagnosis of any other disease that is not a manifestation of Leukocyte Adhesion Deficiency Type II.'}, 'identificationModule': {'nctId': 'NCT03354533', 'briefTitle': 'Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II', 'organization': {'class': 'INDUSTRY', 'fullName': 'Orpha Labs'}, 'officialTitle': 'Study of ORL-1F (L-fucose) in Patients With Leukocyte Adhesion Deficiency Type II', 'orgStudyIdInfo': {'id': 'Ladtwo-1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Treatment with ORL-1F - L-fucose', 'interventionNames': ['Drug: L-fucose']}], 'interventions': [{'name': 'L-fucose', 'type': 'DRUG', 'description': 'Oral ORL-1F', 'armGroupLabels': ['Treatment with ORL-1F - L-fucose']}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Orpha Labs', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}