[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"AAVantgarde Bio Srl\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":76},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,47],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":30,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100605363","phase-1-a-study-of-aavb-039-in-participants-with-stargardt-disease-stgd1-100605363",false,"NCT07161544","A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)","An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)","CELESTE","Inclusion Criteria:\n\n* Molecular diagnosis of Stargardt disease due to ABCA4 mutation\n* Willingness to adhere to protocol per informed consent\n\nExclusion Criteria:\n\n* Unwillingness to meet the requirements of the study\n* Participation in a clinical study with another Investigation Medicinal Product\n* Previous participation in another gene or cell therapy trial\n* Any condition that would preclude subretinal surgery\n* Complicating ocular and\u002For systemic diseases","ALL","8 Years","55 Years",{"count":21,"type":22},75,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.",[29],"Stargardt Disease",[29,31,32,33],"Biallelic Mutation","Gene Mutation","ABCA4","RECRUITING","2026-06-19",{"date":37,"type":38},"2026-06-23","ACTUAL",{"date":40,"type":38},"2025-09-29",{"date":42,"type":22},"2032-07",{"name":44,"class":45},"AAVantgarde Bio Srl","INDUSTRY",8,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":23,"phases":58,"briefSummary":59,"conditions":60,"keywords":62,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":70,"completionDateStruct":72,"leadSponsor":74,"locationsCount":75},"100561563","phase-1-study-of-subretinally-injected-aavb-081-in-patients-with-usher-syndrome-type-ib-ush1b-retinitis-pigmentosa-100561563","NCT06591793","Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa","A Phase 1\u002F2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Subjects With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa","Inclusion Criteria:\n\n* Molecular diagnosis of USB1B due to MYO7A mutation\n* Willingness to adhere to protocol per informed consent\n\nExclusion Criteria:\n\n* Unwillingness to meet the requirements of the study\n* Participation in a clinical study with an Investigation Product in the past 6 months\n* Previous participation in another Gene Therapy trial\n* Any condition that would preclude subretinal surgery\n* Complicating ocular and\u002For systemic diseases","18 Years","50 Years",{"count":57,"type":22},15,[25,26],"The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene. The study will also assess the initial efficacy following AAVB-081 administration.",[61],"Usher Syndrome, Type 1B",[63,64,65,66],"Usher Syndrome","MYO7A","USH1B","Retinitis Pigmentosa","2025-05-01",{"date":69,"type":38},"2025-05-02",{"date":71,"type":38},"2024-07-02",{"date":73,"type":22},"2029-07",{"name":44,"class":45},3,""]