[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07161544":3,"trial-entities:NCT07161544":207,"trial-summary:NCT07161544":211},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":24,"primary_purpose":25,"phases":26,"enrollment_info":29,"interventions":32,"primary_outcomes":42,"secondary_outcomes":46,"sex":55,"minimum_age":56,"maximum_age":57,"healthy_volunteers":58,"eligibility_criteria":59,"std_ages":70,"locations":73,"central_contacts":199,"overall_officials":204,"references":205,"see_also_links":206},"NCT07161544","039-101","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)","RECRUITING","2032-07","2026-07","2026-07-30","2025-09-29","AAVantgarde Bio Srl","INDUSTRY",true,"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.",null,[19],"Stargardt Disease",[19,21,22,23],"Biallelic Mutation","Gene Mutation","ABCA4","INTERVENTIONAL","TREATMENT",[27,28],"PHASE1","PHASE2",{"count":30,"type":31},75,"ESTIMATED",[33],{"type":34,"name":35,"description":36,"armGroupLabels":37},"BIOLOGICAL","AAVB-039","Single subretinal administration",[38,39,40,41],"Cohort 1","Cohort 2","Cohort 3","Cohorts 4-5",[43],{"measure":44,"timeFrame":45},"To measure the number and severity of treatment related adverse events following treatment with AAVB-039","0-60 months",[47,49,51,53],{"measure":48,"timeFrame":45},"Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039",{"measure":50,"timeFrame":45},"Change in ellipsoid zone (EZ) loss following treatment with AAVB-039",{"measure":52,"timeFrame":45},"Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039",{"measure":54,"timeFrame":45},"Change in visual acuity following treatment with AAVB-039","ALL","8 Years","55 Years",false,{"inclusion":60,"exclusion":63,"raw_text":69},[61,62],"Molecular diagnosis of Stargardt disease due to ABCA4 mutation","Willingness to adhere to protocol per informed consent",[64,65,66,67,68],"Unwillingness to meet the requirements of the study","Participation in a clinical study with another Investigation Medicinal Product","Previous participation in another gene or cell therapy trial","Any condition that would preclude subretinal surgery","Complicating ocular and\u002For systemic diseases","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",[71,72],"CHILD","ADULT",[74,91,108,122,137,148,161,175,190],{"facility":75,"status":8,"city":76,"state":77,"zip":78,"country":79,"contacts":80,"geoPoint":88},"Associated Retina Consultants","Phoenix","Arizona","85020","United States",[81,85],{"name":82,"role":83,"email":84},"Study Team","CONTACT","hello@doctrials.com",{"name":86,"role":87},"Dr. Benjamin Bakall","PRINCIPAL_INVESTIGATOR",{"lat":89,"lon":90},33.44838,-112.07404,{"facility":92,"status":8,"city":93,"state":94,"zip":95,"country":79,"contacts":96,"geoPoint":105},"Retina Vitreous Associates Medical Group","Los Angeles","California","90211",[97,100,103],{"name":98,"role":83,"email":99},"Kristina Passoian, Trials Director","Kpassoian@laretina.com",{"name":101,"role":83,"email":102},"Study Referrals","Studyreferrals@laretina.com",{"name":104,"role":87},"Dr. David Liao",{"lat":106,"lon":107},34.05223,-118.24368,{"facility":109,"status":8,"city":110,"state":111,"zip":112,"country":79,"contacts":113,"geoPoint":119},"University of Florida Health Science Center","Jacksonville","Florida","32209",[114,117],{"name":115,"role":83,"email":116},"G. Shabbir Hamdani","Shabbir.hamdani@ufhealth.org",{"name":118,"role":87},"Dr. Sandeep Grover",{"lat":120,"lon":121},30.33218,-81.65565,{"facility":123,"status":8,"city":124,"state":125,"zip":126,"country":79,"contacts":127,"geoPoint":134},"Mayo Clinic","Rochester","Minnesota","55905",[128,132],{"name":129,"role":83,"phone":130,"email":131},"Suzy Wernimont","507-538-8119","Wernimont.Suzanne@mayo.edu",{"name":133,"role":87},"Dr. Brittni Scruggs",{"lat":135,"lon":136},44.02163,-92.4699,{"facility":138,"status":8,"city":139,"state":140,"zip":141,"country":79,"contacts":142,"geoPoint":145},"Retina Consultants of Texas","Bellaire","Texas","77401",[143],{"name":144,"role":87},"Dr. Kenneth Fan",{"lat":146,"lon":147},29.70579,-95.45883,{"facility":149,"status":8,"city":150,"state":140,"zip":151,"country":79,"contacts":152,"geoPoint":158},"Retina Foundation of the Southwest","Dallas","75231",[153,156],{"name":154,"role":83,"email":155},"Senior Clinical Research Coordinator","jclark@retinafoundation.org",{"name":157,"role":87},"Dr. Karl Csaky",{"lat":159,"lon":160},32.78306,-96.80667,{"facility":162,"status":8,"city":163,"state":164,"zip":165,"country":79,"contacts":166,"geoPoint":172},"University of Utah","Salt Lake City","Utah","84112",[167,170],{"name":168,"role":83,"email":169},"Kimberley Wegner Lead Coordinator","Kimberley.wegner@hsc.utah.edu",{"name":171,"role":87},"Dr. Paul Bernstein",{"lat":173,"lon":174},40.76078,-111.89105,{"facility":176,"status":8,"city":177,"state":178,"zip":179,"country":180,"contacts":181,"geoPoint":187},"Moorfields Eye Hospital NHS Foundation Trust","London","England","EC1V 2PD","United Kingdom",[182,185],{"name":183,"role":83,"email":184},"Mithusa Pirabakaran","m.pirabakaran@nhs.net",{"name":186,"role":87},"Prof. Michel Michaelides",{"lat":188,"lon":189},51.50853,-0.12574,{"facility":191,"status":8,"city":177,"state":178,"zip":192,"country":180,"contacts":193,"geoPoint":198},"The Retina Clinic","W1G 9AY",[194,196],{"role":83,"email":195},"ctenquiries@theretinacliniclondon.com",{"name":197,"role":87},"Prof. Paulo-Eduardo Stanga",{"lat":188,"lon":189},[200],{"name":201,"role":83,"phone":202,"email":203},"AAVantgarde Clinical Operations Lead","+448000465680","clinicaltrials@aavantgarde.com",[],[],[],{"nct_id":4,"conditions":208,"biomarkers":209},[19],[210],"Retinal-Specific Phospholipid-Transporting ATPase ABCA4",{"nct_id":4,"found":15,"summary":212,"prompt_version":222},{"design":213,"status":214,"heading":215,"summary":216,"follow_up":217,"word_count":218,"commitments":219,"compensation":220,"drugs_mentioned":221},"This is an interventional study planning to enroll about 75 participants. It will evaluate a single treatment of AAVB-039.","completed","A Study of AAVB-039 for Stargardt Disease","This study is testing a treatment called AAVB-039 for people with Stargardt disease (STGD1). Stargardt disease is an inherited eye condition that causes progressive vision loss. Researchers want to see how safe AAVB-039 is and if people can tolerate it after a single injection into the eye. They will also look for early signs of how well it works. You might be able to join if you are between 8 and 55 years old and have a genetic diagnosis of Stargardt disease due to a specific gene change (ABCA4 mutation). The study plans to enroll about 75 participants, but its current recruitment status is unclear.","Participants will be followed for treatment-related side effects for up to 60 months (5 years) after receiving AAVB-039.",105,"Not specified in the trial record.","Not stated in the trial record.",[35],"v2"]