[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"stargardt-disease-1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:stargardt-disease-1":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,54,89],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":28,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100550303","prescreening-study-to-identify-potential-stargardt-participants-for-acdn-01-clinical-trials-starpath-100550303",false,"NCT06445322","Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)","Prescreening Study to Identify Potential Participants With ABCA4-related Retinopathy for ACDN-01 Clinical Trials","Key Inclusion Criteria:\n\n* Presence of mutations in the ABCA4 gene\n* ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)\n\nKey Exclusion Criteria:\n\n* The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy\n* Retinal disease other than ABCA4-related retinopathy\n* Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.","ALL","5 Years",{"count":19,"type":20},50,"ESTIMATED","OBSERVATIONAL","This is an observational prescreening study. Individuals who are eligible for prescreening will undergo testing procedures that may be used to determine eligibility in ACDN-01 clinical trials.",[24,25,26,27],"Stargardt Disease","Stargardt Disease 1","Cone Rod Dystrophy","Juvenile Macular Degeneration",[29,30,24,31,32,33,34,35,36,37,38,39,40],"ABCA4","ABCA4-related retinopathy","Stargardt macular dystrophy","Cone rod dystrophy","Gene editing","RNA","Gene Therapy","Exon editing","IRD","Inherited retinal disease","Inherited retinal dystrophy","Inherited retinal degeneration","RECRUITING","2026-03-09",{"date":44,"type":45},"2026-03-11","ACTUAL",{"date":47,"type":45},"2024-06-20",{"date":49,"type":20},"2030-08-31",{"name":51,"class":52},"Ascidian Therapeutics, Inc","INDUSTRY",8,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":4,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":16,"minAge":61,"maxAge":4,"enrollmentInfo":62,"targetDuration":4,"studyType":64,"phases":65,"briefSummary":68,"conditions":69,"keywords":71,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":79,"lastUpdatePostDateStruct":80,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":88},"100593127","phase-1-safety-and-preliminary-efficacy-of-vg801-in-patients-with-abca4-mutation-associated-retinal-dystrophy-stargardt-disease-100593127","NCT07002398","Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)","A Single Arm, Ph1\u002F2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)","Inclusion Criteria:\n\nTo be eligible for study entry, subjects must satisfy all the following criteria:\n\n1. Written informed consent.\n2. Subjects aged ≥ 6 years.\n3. Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).\n4. Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).\n5. Poor vision in the study eye.\n\nExclusion Criteria:\n\nSubjects will be excluded from the study if one or more of the following statements are applicable to either eye:\n\n1. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.\n2. Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.\n3. History of intraocular surgery within the previous 6 months.\n4. Previous participation in a gene therapy trial.\n5. Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.\n6. Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).","6 Years",{"count":63,"type":20},15,"INTERVENTIONAL",[66,67],"PHASE1","PHASE2","This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.",[70,25],"Retinal Dystrophy Due to Biallelic ABCA4 Mutations",[72,73,74,75,76,77,78],"ABCA4 mutation","Gene therapy","Eye disease","Stargardt disease","Retinal disease","Retinal degeneration","VG801","2025-12-04",{"date":81,"type":45},"2025-12-11",{"date":83,"type":45},"2024-12-23",{"date":85,"type":20},"2026-05",{"name":87,"class":52},"VeonGen Therapeutics GmbH",1,{"id":90,"slug":91,"hasResults":11,"nctId":92,"briefTitle":93,"officialTitle":94,"acronym":4,"eligibilityCriteria":95,"healthyVolunteers":11,"sex":16,"minAge":96,"maxAge":4,"enrollmentInfo":97,"targetDuration":4,"studyType":64,"phases":98,"briefSummary":99,"conditions":100,"keywords":101,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":102,"lastUpdatePostDateStruct":103,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":110},"100551996","phase-1-study-to-evaluate-acdn-01-in-abca4-related-stargardt-retinopathy-stellar-100551996","NCT06467344","Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)","ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy","Key Inclusion Criteria:\n\n* Presence of mutations in the ABCA4 gene\n* ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)\n* Area of atrophy located in the macula of the study eye\n* BCVA of 20\u002F50 (0.4 logMAR) or worse\n\nKey Exclusion Criteria:\n\n* The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy\n* Retinal disease other than ABCA4-related retinopathy\n* Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.","12 Years",{"count":63,"type":20},[66,67],"This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.",[24,26,27,25],[29,30,24,31,32,33,34,35,36,37,38,39,40],"2025-11-25",{"date":104,"type":45},"2025-12-02",{"date":106,"type":45},"2024-06-11",{"date":108,"type":20},"2030-12-01",{"name":51,"class":52},10]