Ignite Creation Date:
2025-12-24 @ 10:52 PM
Ignite Modification Date:
2025-12-25 @ 8:21 PM
Study NCT ID:
NCT03404869
Status:
UNKNOWN
Last Update Posted:
2019-01-25
First Post:
2018-01-13
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Study of ORL-1M (D-mannose) in Patients With CDG-Ib
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C535740', 'term': 'Congenital disorder of glycosylation type 1B'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 5}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2015-03-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-01', 'completionDateStruct': {'date': '2019-06-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2019-01-22', 'studyFirstSubmitDate': '2018-01-13', 'studyFirstSubmitQcDate': '2018-01-13', 'lastUpdatePostDateStruct': {'date': '2019-01-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-01-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-06-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Improvement in hypoglycemia, diarrhea and vomiting.', 'timeFrame': '6 months after treatment started', 'description': 'Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.'}], 'secondaryOutcomes': [{'measure': 'Improved glycosylation pattern of serum transferrin.', 'timeFrame': '30 days after treatment started', 'description': 'Normalized isoelectric focusing pattern of serum transferrin.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False}, 'conditionsModule': {'keywords': ['CDGIb MANNOSEPHOSPHATE ISOMERASE DEFICIENCY MPI DEFICIENCY PROTEIN-LOSING ENTEROPATHY-HEPATIC FIBROSIS SYNDROME SAGUENAY-LAC SAINT-JEAN SYNDROME SLSJ SYNDROME'], 'conditions': ['CDG Ib']}, 'referencesModule': {'references': [{'pmid': '35120836', 'type': 'DERIVED', 'citation': 'Rebelo AL, Chevalier MT, Russo L, Pandit A. Role and therapeutic implications of protein glycosylation in neuroinflammation. Trends Mol Med. 2022 Apr;28(4):270-289. doi: 10.1016/j.molmed.2022.01.004. Epub 2022 Feb 1.'}]}, 'descriptionModule': {'briefSummary': 'Study of ORL-1M in Patients With CDG-Ib'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of CGD-1b.\n* Less than 18 years old.\n\nExclusion Criteria:\n\n* Diagnosis of any other disease that is not a manifestation of CGD-1b.'}, 'identificationModule': {'nctId': 'NCT03404869', 'briefTitle': 'Study of ORL-1M (D-mannose) in Patients With CDG-Ib', 'organization': {'class': 'INDUSTRY', 'fullName': 'Orpha Labs'}, 'officialTitle': 'Study of ORL-1M (D-mannose) in Patients With CDG-Ib', 'orgStudyIdInfo': {'id': 'PMID-1'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'OTHER', 'label': 'Treatment with ORL-1M - D-mannose', 'interventionNames': ['Drug: ORL-1M - D-mannose']}], 'interventions': [{'name': 'ORL-1M - D-mannose', 'type': 'DRUG', 'description': 'Oral ORL-1M', 'armGroupLabels': ['Treatment with ORL-1M - D-mannose']}]}, 'contactsLocationsModule': {'locations': [{'zip': '06100', 'city': 'Ankara', 'status': 'RECRUITING', 'country': 'Turkey (Türkiye)', 'contacts': [{'name': 'Study Coordinator Study Coordinator', 'role': 'CONTACT', 'email': 'study.coordinator@orphalabs.com', 'phone': '+905377636241'}], 'facility': 'Orpha Labs', 'geoPoint': {'lat': 39.91987, 'lon': 32.85427}}], 'centralContacts': [{'name': 'Study Coordinator Study Coordinator', 'role': 'CONTACT', 'email': 'business.development@orphalabs.com', 'phone': '+90 537 763 6241'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Orpha Labs', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}