Ignite Creation Date:
2026-03-26 @ 3:19 PM
Ignite Modification Date:
2026-03-31 @ 12:50 PM
Study NCT ID:
NCT07485660
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-03-20
First Post:
2026-03-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Care Pathway for Patients With Fabry's Disease (Fabry-PATH)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2026-03-25'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}], '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': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'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'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 200}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-04', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2026-03', 'completionDateStruct': {'date': '2026-11', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2026-03-16', 'studyFirstSubmitDate': '2026-03-16', 'studyFirstSubmitQcDate': '2026-03-16', 'lastUpdatePostDateStruct': {'date': '2026-03-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2026-03-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': "Diagnostic time in care pathways between men and women with Fabry's disease", 'timeFrame': '8 months', 'description': 'The average diagnostic time, compared between men and women, and the diagnostic methods (entry point into the disease), compared according to sex.'}, {'measure': 'Comparison of diagnostic methods between men and women', 'timeFrame': '8 months', 'description': 'Data collection at the point of entry into the disease: via symptoms or family screening'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Fabry', 'Care pathway', 'Family screening', 'Symptoms', 'Comparison according to gender'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': "Fabry's disease is an X-linked disorder that manifests differently in men and women, leading to differences in healthcare pathways. In women, the disease is sometimes diagnosed through family screening despite the presence of symptoms (symptoms appear later than in men, and the phenotypic expression of the disease is sometimes more subtle). Conversely, in men, the presence of symptoms combined with abnormalities in medical examinations facilitates diagnosis. There is limited data on the differences in healthcare pathways between men and women, which could nevertheless impact diagnosis by identifying a profile of at-risk patients, and consequently, their management.\n\nThe goal of this present study is to describe the differences in care pathways between men and women with Fabry's disease, distinguishing entry via symptoms or family screening through a non-interventional study based on a questionnaire sent to patients and on the collection of medical data at the time of diagnosis."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': "Any adult patients with a Fabry's disease diagnosis", 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Adult patient diagnosed with Fabry's disease\n\nExclusion Criteria:\n\n* Minor patients"}, 'identificationModule': {'nctId': 'NCT07485660', 'acronym': 'Fabry-PATH', 'briefTitle': "Care Pathway for Patients With Fabry's Disease (Fabry-PATH)", 'organization': {'class': 'OTHER', 'fullName': 'University Hospital, Toulouse'}, 'officialTitle': "Care Pathway for Patients With Fabry's Disease (Fabry-PATH)", 'orgStudyIdInfo': {'id': 'RC31/26/0050'}, 'secondaryIdInfos': [{'id': '2026-A00322-49', 'type': 'OTHER', 'domain': 'ANSM'}]}, 'armsInterventionsModule': {'armGroups': [{'label': "Fabry's disease patients", 'description': "Male and female patients with Fabry's disease diagnosed by the presence of symptoms or following family screening", 'interventionNames': ['Other: Self-questionnaire']}], 'interventions': [{'name': 'Self-questionnaire', 'type': 'OTHER', 'description': "Medical data of Fabry's disease", 'armGroupLabels': ["Fabry's disease patients"]}]}, 'contactsLocationsModule': {'locations': [{'zip': '31400', 'city': 'Toulouse', 'state': 'Occitanie', 'country': 'France', 'contacts': [{'name': 'Olivier LAIREZ, MD', 'role': 'CONTACT', 'email': 'lairez.o@chu-toulouse.fr', 'phone': '561323072', 'phoneExt': '+33'}], 'facility': 'Service de Cardiologie - 1 Avenue du Professeur Jean Poulhès', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}], 'centralContacts': [{'name': 'Olivier LAIREZ', 'role': 'CONTACT', 'email': 'lairez.o@chu-toulouse.fr', 'phone': '+33561323072'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Hospital, Toulouse', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}