Ignite Creation Date:
2025-12-24 @ 2:55 PM
Ignite Modification Date:
2025-12-26 @ 8:29 AM
Study NCT ID:
NCT05629559
Status:
RECRUITING
Last Update Posted:
2025-02-21
First Post:
2022-11-04
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
4D-310 in Adults With Fabry Disease and Cardiac Involvement
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}, {'id': 'D016464', 'term': 'Lysosomal Storage 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': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D008064', 'term': 'Lipidoses'}], 'ancestors': [{'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': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 18}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-10-16', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-02', 'completionDateStruct': {'date': '2030-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-02-19', 'studyFirstSubmitDate': '2022-11-04', 'studyFirstSubmitQcDate': '2022-11-17', 'lastUpdatePostDateStruct': {'date': '2025-02-21', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-11-29', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence and severity of adverse events', 'timeFrame': '1 year', 'description': 'Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose'}]}, 'oversightModule': {'isUsExport': True, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Lysosomal Storage Diseases', 'Nervous System Brain Diseases', 'Metabolic', 'Inborn Brain Diseases', 'Central Nervous System Diseases', 'Nervous System Diseases', 'Cerebral Small Vessel Diseases', 'Cerebrovascular Disorders', 'Vascular Diseases', 'Cardiovascular Diseases', 'Genetic Diseases', 'X-Linked', 'Inborn', 'Metabolic Diseases', 'Lipid Metabolism Disorders', 'Sphingolipidoses', 'Metabolism', 'Inborn Errors', 'Lipidoses', 'Lipid Metabolism'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': 'This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement', 'detailedDescription': 'This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Male or female ≥ 18 to ≤65 years of age\n2. Pathogenic GLA mutation consistent with Fabry Disease\n3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement\n4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat\n5. Individuals receiving ERT, must be on a stable dose for at least 6 months\n6. Agree to use highly effective contraception\n\nExclusion Criteria:\n\n1. Presence of pre-existing antibodies to 4D-310 capsid or to AGA\n2. eGFR \\<65 mL/min/1.73 m2\n3. Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis\n4. HIV, active or chronic hepatitis B or C,\n5. Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control\n6. History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)\n7. Contraindication to systemic corticosteroid therapy or immunosuppressive therapy\n8. Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.\n9. Moderately severe to severe cardiovascular disease or uncontrolled hypertension\n10. Left ventricular ejection fraction of \\<45% on echocardiogram (ECHO)\n11. Currently receiving investigational drug, device or therapy or having ever received gene therapy\n12. History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation\n13. History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)\n14. Pregnant or breast-feeding\n\nNote: Other inclusion and exclusion criteria apply'}, 'identificationModule': {'nctId': 'NCT05629559', 'briefTitle': '4D-310 in Adults With Fabry Disease and Cardiac Involvement', 'organization': {'class': 'INDUSTRY', 'fullName': '4D Molecular Therapeutics'}, 'officialTitle': 'An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement', 'orgStudyIdInfo': {'id': '4D-310-C002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '4D-310 Dose Level -1', 'description': 'Single IV administration of 4D-310 Dose Level -1', 'interventionNames': ['Biological: 4D-310']}, {'type': 'EXPERIMENTAL', 'label': '4D-310 Dose Level 2', 'description': 'Single IV administration of 4D-310 Dose Level 2', 'interventionNames': ['Biological: 4D-310']}, {'type': 'EXPERIMENTAL', 'label': '4D-310 Dose Level 3', 'description': 'Single IV administration of 4D-310 at Dose Level 3', 'interventionNames': ['Biological: 4D-310']}, {'type': 'EXPERIMENTAL', 'label': '4D-310 Dose Level 1 (No longer enrolling)', 'description': 'No longer enrolling - Single IV administration of 4D-310 at Dose Level 1', 'interventionNames': ['Biological: 4D-310']}], 'interventions': [{'name': '4D-310', 'type': 'BIOLOGICAL', 'description': 'Single IV administration of 4D-310', 'armGroupLabels': ['4D-310 Dose Level -1', '4D-310 Dose Level 1 (No longer enrolling)', '4D-310 Dose Level 2', '4D-310 Dose Level 3']}]}, 'contactsLocationsModule': {'locations': [{'zip': '3050', 'city': 'Melbourne', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'Australia', 'facility': 'Royal Melbourne Hospital', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'zip': '6000', 'city': 'Perth', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'Australia', 'facility': 'Royal Perth Hospital', 'geoPoint': {'lat': -31.95224, 'lon': 115.8614}}, {'zip': '2145', 'city': 'Westmead', 'status': 'NOT_YET_RECRUITING', 'country': 'Australia', 'contacts': [{'name': '4DMT Patient Advocacy', 'role': 'CONTACT', 'email': 'clinicaltrials@4DMT.com', 'phone': '(888) 748-8881'}], 'facility': 'Westmead Hospital', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '11217', 'city': 'Taipei', 'status': 'RECRUITING', 'country': 'Taiwan', 'contacts': [{'name': '4DMT Patient Advocacy', 'role': 'CONTACT', 'email': 'clinicaltrials@4DMT.com', 'phone': '(888) 748-8881'}], 'facility': 'Taipei Veterans General Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}], 'centralContacts': [{'name': '4DMT Patient Advocacy', 'role': 'CONTACT', 'email': 'clinicaltrials@4DMT.com', 'phone': '(888) 748-8881'}], 'overallOfficials': [{'name': 'Alan H Cohen, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': '4D Molecular Therapeutics'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': '4D Molecular Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}