Ignite Creation Date:
2025-12-25 @ 12:21 AM
Ignite Modification Date:
2026-01-02 @ 5:44 AM
Study NCT ID:
NCT06949358
Status:
COMPLETED
Last Update Posted:
2025-04-29
First Post:
2025-04-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009542', 'term': 'Niemann-Pick Diseases'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D015616', 'term': 'Histiocytosis, Non-Langerhans-Cell'}, {'id': 'D015614', 'term': 'Histiocytosis'}, {'id': 'D008206', 'term': 'Lymphatic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000608782', 'term': 'olipudase alfa'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-11-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-04', 'completionDateStruct': {'date': '2025-04-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-04-22', 'studyFirstSubmitDate': '2025-04-22', 'studyFirstSubmitQcDate': '2025-04-22', 'lastUpdatePostDateStruct': {'date': '2025-04-29', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-04-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-04-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of adverse events (AEs) / serious adverse events (SAEs)', 'timeFrame': 'Baseline to approximately 5 years'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Niemann-Pick Disease']}, 'descriptionModule': {'briefSummary': "This is an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632 Study in France until olipudase alfa reimbursement is granted in France.\n\nStudy and treatment duration:\n\nThe period between the patient's completion of Study DFI12712 or LTS13632 and olipudase alfa reimbursement is available in France.\n\nIn case reimbursement will not be obtained, this study will end 5 years after starting.\n\nVisit frequency: every 2 weeks."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* The patient has completed Study DFI12712 (ASCEND) or LTS13632 in France\n* The patient must provide signed, informed consent prior to performing any study-related procedures.\n* The patient is willing to comply with the clinical protocol.\n* The patient, if female and of childbearing potential, must have a negative pregnancy test result \\[urine beta-human chorionic gonadotropin (β-HCG)\\] at enrollment.\n* Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception for the entire duration of the treatment period and for at least 28 days after receiving the last study drug dose.\n\nExclusion Criteria:\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n* Any patient who has not participated in the Study DFI12712 or the Study LTS13632\n* A patient who experienced any systemic hypersensitivity reactions to olipudase alfa in Study DFI12712 or Study LTS13632 which, in the opinion of the Investigator, could indicate that treatment continuation may present an unreasonable risk.\n* The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.\n* The patient is unwilling or unable to abstain from alcohol for 1 day prior to and 3 days after each olipudase alfa infusion for the duration of the treatment period.\n* Individuals accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalized.\n* The patient is concurrently participating in another clinical study of investigational treatment.\n* Any of the following medical conditions:\n* The patient has any new condition or worsening of an existing condition which, in the opinion of the Investigator, would make the patient unsuitable for enrollment or could interfere with the patient's participating in or completing the study.\n* Requirement for recurrent dose adjustment of anticoagulation treatment over the last 6 months.\n* Pregnancy or breastfeeding.\n\nThe above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial."}, 'identificationModule': {'nctId': 'NCT06949358', 'briefTitle': 'A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sanofi'}, 'officialTitle': 'A Long-Term Follow-up Study to Evaluate Safety and Tolerability of Olipudase Alfa in Patients Who Completed the DFI12712 or the LTS13632 Study in France', 'orgStudyIdInfo': {'id': 'PTA17397'}, 'secondaryIdInfos': [{'id': '2024-515304-39', 'type': 'REGISTRY', 'domain': 'CTIS'}, {'id': '2021-004109-39', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'GZ402665', 'description': 'Olipudase alfa administered intravenously every 2 weeks', 'interventionNames': ['Drug: Olipudase alfa']}], 'interventions': [{'name': 'Olipudase alfa', 'type': 'DRUG', 'otherNames': ['GZ402665'], 'description': 'Pharmaceutical form:Powder for concentrate for solution for infusion-Route of administration:intravenous infusion', 'armGroupLabels': ['GZ402665']}]}, 'contactsLocationsModule': {'locations': [{'zip': '69500', 'city': 'Bron', 'country': 'France', 'facility': 'Investigational Site Number : 2500002', 'geoPoint': {'lat': 45.73865, 'lon': 4.91303}}, {'zip': '75020', 'city': 'Paris', 'country': 'France', 'facility': 'Investigational Site Number : 2500001', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'YES', 'description': "Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org"}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sanofi', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}