Ignite Creation Date:
2025-12-24 @ 5:23 PM
Ignite Modification Date:
2025-12-25 @ 2:58 PM
Study NCT ID:
NCT06880250
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-03-17
First Post:
2025-03-11
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy and Safety of Enzyme Replacement Therapy in Patients with Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, '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': 'OTHER', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-03', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-03-11', 'studyFirstSubmitDate': '2025-03-11', 'studyFirstSubmitQcDate': '2025-03-11', 'lastUpdatePostDateStruct': {'date': '2025-03-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-03-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change from Baseline in Lyso-Gb3 Level', 'timeFrame': 'From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation', 'description': 'Level of globotriaosylsphingosine (lyso-Gb3) is measured in dried blood spots.'}], 'secondaryOutcomes': [{'measure': 'Change from Baseline in α-GAL Activity', 'timeFrame': 'From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation', 'description': 'Level of α-Galactosidase (α-GAL) activity is measured in dried blood spots.'}, {'measure': 'Change from Baseline in GFR', 'timeFrame': 'From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation', 'description': 'Glomerular filtration rate (GFR) is measured by methods available in routine medical use.'}, {'measure': 'Change from Baseline in Neuropathic Pain Level', 'timeFrame': 'From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation', 'description': 'Neuropathic pain is assessed according to verbal scale: no pain, mild pain, moderate pain, severe pain, very severe pain, intolerable pain. Corresponding value of 0, 2, 4, 6, 8, 10 is assigned.'}, {'measure': 'Frequency of Significant Clinical Outcomes', 'timeFrame': 'From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation', 'description': 'Significant clinical outcomes include cardiac rhythm disturbances (atrial fibrillation, persistent supraventricular or ventricular tachycardia), heart failure, stroke, initiation of renal replacement therapy and death from any cause.'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Fabry', 'ERT', 'enzyme replacement therapy'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': 'The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is:\n\nWhat medical problems do participants have?\n\nParticipants already on enzyme replacement therapy as part of their regular medical care for Fabry disease will be observed as usual. Their data will be collected by their doctor.', 'detailedDescription': 'All patients on enzyme replacement therapy could be enrolled in the study. Three medicines are currently available in Russia (Fabagal®, Fabrazyme®, Replagal®) so the patients form three cohorts.\n\nFor all patients retrospective data are collected for 12 months prior enrollment.\n\nAll patients are observed prospectively for 6 months. At the point of 6 months patients taking Fabrazyme or Replagal at the moment finish the study.\n\nPatients on Fabagal are observed for 12 more months (up to 18 months after enrollment).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '8 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Russian patients', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Men and women with a confirmed diagnosis of Fabry disease;\n* Prescribed course of enzyme replacement therapy with any drug (FABAGAL, FABRAZIM, REPLAGAL);\n* Age 8 - 65 years;\n* Signed informed consent of the patient and/or his/her legal representative for participation in the study and processing of personal data.\n\nExclusion Criteria:\n\n* Renal replacement therapy or kidney transplantation;\n* Critical illness unrelated to Fabry disease;\n* Pregnancy or breastfeeding;\n* Refusal of the patient (or his/her legal representative) from further participation in the study or medical care.'}, 'identificationModule': {'nctId': 'NCT06880250', 'acronym': 'FAVORIT', 'briefTitle': 'Efficacy and Safety of Enzyme Replacement Therapy in Patients with Fabry Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'NPO Petrovax'}, 'officialTitle': 'Open-label Comparative Cohort Non-interventional Epidemiological Study of the Efficacy and Safety of Enzyme Replacement Therapy in Russian Patients with Fabry Disease', 'orgStudyIdInfo': {'id': 'FAVORIT'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Fabagal', 'description': 'Patients taking Fabagal® as the enzyme replacement therapy'}, {'label': 'Fabrazyme', 'description': 'Patients taking Fabrazyme® as the enzyme replacement therapy'}, {'label': 'Replagal', 'description': 'Patients taking Replagal® as the enzyme replacement therapy'}]}, 'contactsLocationsModule': {'centralContacts': [{'name': 'Marat Dautov', 'role': 'CONTACT', 'email': 'info@petrovax.ru', 'phone': '+7 (495) 730-75-45'}], 'overallOfficials': [{'name': 'Sergey V. Moiseev, MD', 'role': 'STUDY_CHAIR', 'affiliation': 'Sechenov First Moscow State Medical University (Sechenov University)'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'NPO Petrovax', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}