Ignite Creation Date:
2025-12-25 @ 3:27 AM
Ignite Modification Date:
2025-12-26 @ 2:07 AM
Study NCT ID:
NCT01494805
Status:
COMPLETED
Last Update Posted:
2017-09-01
First Post:
2011-12-14
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D008268', 'term': 'Macular Degeneration'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D015861', 'term': 'Retinal Neovascularization'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D057135', 'term': 'Wet Macular Degeneration'}, {'id': 'D012164', 'term': 'Retinal Diseases'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D009389', 'term': 'Neovascularization, Pathologic'}, {'id': 'D008679', 'term': 'Metaplasia'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D000069579', 'term': 'Ranibizumab'}], 'ancestors': [{'id': 'D061067', 'term': 'Antibodies, Monoclonal, Humanized'}, {'id': 'D000911', 'term': 'Antibodies, Monoclonal'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 40}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-08', 'completionDateStruct': {'date': '2017-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-08-30', 'studyFirstSubmitDate': '2011-12-14', 'studyFirstSubmitQcDate': '2011-12-15', 'lastUpdatePostDateStruct': {'date': '2017-09-01', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2011-12-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2017-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection', 'timeFrame': 'Primary endpoint at 1 month', 'description': '1. Ocular examination:\n\n * Ocular inflammation\n * Intraocular pressure\n * Visual acuity\n * Retinal bleeding\n2. Abnormal laboratory data'}], 'secondaryOutcomes': [{'measure': 'Maintenance or improvement of vision without the necessity of ranibizumab re-injections', 'timeFrame': 'Up to 3 years', 'description': '1. Best-corrected visual acuity\n2. CNV lesion\n3. Foveal thickness'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Age-related Macular Degeneration', 'AAV', 'AMD', 'Wet AMD', 'Neovascular AMD', 'Gene Therapy', 'Ocular', 'Ocular Gene Therapy', 'Eye diseases', 'Macular Degeneration', 'Retinal Degeneration', 'Retinal Neovascularization', 'Wet Macular Degeneration', 'Retinal Diseases'], 'conditions': ['Macular Degeneration', 'Age-related Maculopathies', 'Age-related Maculopathy', 'Maculopathies,Age-related', 'Maculopathy,Age-related', 'Retinal Degeneration', 'Retinal Neovascularization', 'Eye Diseases']}, 'referencesModule': {'references': [{'pmid': '27865764', 'type': 'DERIVED', 'citation': 'Constable IJ, Pierce CM, Lai CM, Magno AL, Degli-Esposti MA, French MA, McAllister IL, Butler S, Barone SB, Schwartz SD, Blumenkranz MS, Rakoczy EP. Phase 2a Randomized Clinical Trial: Safety and Post Hoc Analysis of Subretinal rAAV.sFLT-1 for Wet Age-related Macular Degeneration. EBioMedicine. 2016 Dec;14:168-175. doi: 10.1016/j.ebiom.2016.11.016. Epub 2016 Nov 10.'}, {'pmid': '26431823', 'type': 'DERIVED', 'citation': 'Rakoczy EP, Lai CM, Magno AL, Wikstrom ME, French MA, Pierce CM, Schwartz SD, Blumenkranz MS, Chalberg TW, Degli-Esposti MA, Constable IJ. Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial. Lancet. 2015 Dec 12;386(10011):2395-403. doi: 10.1016/S0140-6736(15)00345-1. Epub 2015 Sep 30.'}]}, 'descriptionModule': {'briefSummary': 'The study will involve approximately 40 subjects aged 55 or above who have exudative age-related macular degeneration (wet AMD). Patients will be randomized to receive one of two doses of rAAV.sFlt-1 or assigned to the control group.', 'detailedDescription': 'A new treatment for exudative age-related macular degeneration (wet AMD) is being investigated. The purpose of this Phase I/II clinical research study is to examine the baseline safety and efficacy of an experimental study drug to treat a complication of the disease which leads to vision loss. The name of the study drug is rAAV.sFlt-1.\n\nThis experimental study uses a non-pathogenic virus to express a therapeutic protein within the eye. The therapeutic diminishes the growth of abnormal blood vessels under the retina. The duration of effect is thought to be long-term (years) following a single administration.\n\nThe clinical research study will look at the baseline safety and efficacy of a single injection of rAAV.sFlt-1 injected directly into the eye.\n\nApproximately forty (40) subjects will participate in Australia. The primary endpoint of the study is at one month, with extended follow up for 3 years.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '55 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age greater than or equal to 55 years;\n* Subfoveal CNV secondary to AMD and with best corrected visual acuity of 3/60 - 6/9 with 6/60 or better in the other eye;\n* Fluorescein angiogram of the study eye must show evidence of a leaking subfoveal choroidal neovascular lesion, or CNV currently under active management with anti-VEGF therapy;\n* Must be a candidate for anti-VEGF intravitreal injections;\n* No previous retinal treatment of photodynamic therapy or laser;\n* Able to provide informed consent;\n* Able to comply with protocol requirements, including follow-up visits.\n\nExclusion Criteria:\n\n* Liver enzymes \\> 2 X upper limit of normal;\n* Any prior treatment for AMD in the study / control eye, excluding anti-VEGF injections;\n* Extensive sub-foveal scarring, extensive geographic atrophy, or thick subretinal blood in the study eye as determined by the investigator;\n* Significant retinal disease other than sub-foveal CNV AMD;'}, 'identificationModule': {'nctId': 'NCT01494805', 'acronym': 'AMD', 'briefTitle': 'Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration', 'organization': {'class': 'OTHER', 'fullName': 'Lions Eye Institute, Perth, Western Australia'}, 'officialTitle': 'A Phase I/II Controlled Dose-escalating Trial to Establish the Baseline Safety and Efficacy of a Single Subretinal Injection of rAAV.sFlt-1 Into Eyes of Patients With Exudative Age-related Macular Degeneration (AMD)', 'orgStudyIdInfo': {'id': '2008-135'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Low Dose rAAV.sFlt-1', 'interventionNames': ['Biological: rAAV.sFlt-1']}, {'type': 'EXPERIMENTAL', 'label': 'High Dose rAAV.sFlt-1', 'interventionNames': ['Biological: rAAV.sFlt-1']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Control - ranibizumab only', 'interventionNames': ['Other: Control (ranibizumab alone)']}], 'interventions': [{'name': 'rAAV.sFlt-1', 'type': 'BIOLOGICAL', 'description': '1 x 10\\^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection', 'armGroupLabels': ['Low Dose rAAV.sFlt-1']}, {'name': 'rAAV.sFlt-1', 'type': 'BIOLOGICAL', 'description': '1 x 10\\^11 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection', 'armGroupLabels': ['High Dose rAAV.sFlt-1']}, {'name': 'Control (ranibizumab alone)', 'type': 'OTHER', 'description': 'Patients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).', 'armGroupLabels': ['Control - ranibizumab only']}]}, 'contactsLocationsModule': {'locations': [{'zip': '6009', 'city': 'Nedlands', 'state': 'Western Australia', 'country': 'Australia', 'facility': 'Lions Eye Institute', 'geoPoint': {'lat': -31.98184, 'lon': 115.8073}}], 'overallOfficials': [{'name': 'Ian Constable, Professor', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Lions Eye Institute'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Lions Eye Institute, Perth, Western Australia', 'class': 'OTHER'}, 'collaborators': [{'name': 'Adverum Biotechnologies, Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Scientific Investigator', 'investigatorFullName': 'Prof. P. Elizabeth Rakoczy', 'investigatorAffiliation': 'Lions Eye Institute, Perth, Western Australia'}}}}