[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Splice Bio\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":78},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,46],{"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":4,"briefSummary":24,"conditions":25,"keywords":30,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100549513","an-observational-study-in-subjects-to-follow-the-progression-of-stargardt-disease-type-1-stgd1-caused-by-bi-allelic-autosomal-recessive-mutations-in-the-abca4-gene-100549513",false,"NCT06435000","An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene","An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene","POLARIS","Inclusion Criteria:\n\n1. Provide written consent\n2. Are male or female aged 12-65 years old\n3. Have a diagnosis of STGD1 caused by bi-allelic likely pathogenic or pathogenic variants in the ABCA4 gene confirmed genotypically by an accredited genotyping laboratory\n4. Have a history of STGD1 progression within the last 2 years, in the opinion of the investigator.\n5. Eligible eye(s) must have:\n\n   1. BCVA of between 24-88 ETDRS letters, inclusive (20\u002F20 - 20\u002F320 Snellen equivalent, 0.0-1.2 logMAR) at the Screening Visit.\n   2. Clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease.\n   3. Fundus autofluorescence (FAF) measurement of definitely decreased autofluorescence (DDAF) as measured by the Central Reading Center (CRC).\n   4. Total lesion must be imaged in its entirety.\n   5. All total lesion borders must be ≥300 microns from all image edges.\n6. Eligible eye(s) must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging.\n\nExclusion Criteria:\n\n1. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.\n2. Have any concurrent ocular disease that would affect study procedures or outcomes (e.g., cataracts; subjects can be enrolled 90 days after successful cataract surgery) in eligible eyes.\n3. Have two likely pathogenic or pathogenic variants (not STGD1) in autosomal recessive inherited retinal dystrophy (IRD) genes or a single likely pathogenic or pathogenic variant in autosomal dominant or X-linked IRD genes.\n4. Have had any intraocular surgery or thermal laser within 90 days of study entry or any prior thermal laser in the macular region within the eligible eye(s).\n5. Have any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the study period.\n6. Are unwilling to stop taking the following products at Screening and throughout the study:\n\n   1. Supplements containing vitamin A or beta-carotene, liver-based products.\n   2. Prescription oral retinoids.\n7. Have actively participated in an investigational therapy study or have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer. Note: any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or intravitreal or sub-retinal injections exclude the subject from study participation.\n8. Have known serious allergies to the fluorescein dye that might be used to measure intraocular pressure (IOP), ocular dilating drops, topical ocular anesthetic, or any history of anaphylaxis reaction.\n9. Have a history of amblyopia in the eligible eye(s).\n10. Have any significant ocular or non-ocular disease\u002Fdisorder (or medication and\u002For laboratory test abnormalities) which, in the opinion of the investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or affect the subject's ability to participate in the study.","ALL","12 Years","65 Years",{"count":21,"type":22},75,"ESTIMATED","OBSERVATIONAL","This is an Observational Study to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene\n\nThis is a multicenter study which will enroll approximately 75 subjects",[26,27,28,29],"Stargardt","Stargardt's Disease","Stargardt Disease","STGD1",[15,31,29,32],"Splicebio","ABCA4","RECRUITING","2025-09-18",{"date":36,"type":37},"2025-09-19","ACTUAL",{"date":39,"type":37},"2024-03-29",{"date":41,"type":22},"2027-02",{"name":43,"class":44},"Splice Bio","INDUSTRY",20,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":51,"acronym":52,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":54,"targetDuration":4,"studyType":56,"phases":57,"briefSummary":60,"conditions":61,"keywords":64,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":76,"locationsCount":77},"100588528","phase-1-a-phase-12-first-in-human-dose-escalationexpansion-study-to-evaluate-the-safety-tolerability-and-preliminary-efficacy-of-a-subretinal-injection-of-sb-007-in-subjects-with-stargardt-disease-stgd1-100588528","NCT06942572","A Phase 1\u002F2, First-in-Human Dose Escalation\u002FExpansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)","A Phase 1\u002F2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation\u002FExpansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene","ASTRA","Inclusion Criteria:\n\nTo be eligible for study participation, subjects must meet the following criteria:\n\n1. Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same.\n2. Are male or female adolescents and adults, aged as follows:\n\n   1. In Part A, subjects will be ≥18 to ≤65\\* years (inclusive)\n   2. In Part B, subject age is planned as ≥12 to ≤65\\* years (inclusive) \\*Subjects aged \\>65 years may be eligible in Parts A and B, following discussion with, and approval by, the Medical Monitor.\n3. Are able to understand and comply with the study procedures.\n4. Have a diagnosis of STGD1 caused by bi-allelic pathogenic, or likely pathogenic, variants in the ABCA4 gene confirmed genotypically by an accredited genetic testing laboratory\n5. Clinical evidence consistent with Stargardt Disease type 1.\n6. For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active treatment, at Day 0.\n7. For both WOCBP and male subjects (or their female partners who are of child-bearing potential), agree to either strict abstinence or, if sexually active, use an acceptable contraception measure for 3 months from Day 0\n8. Must have clear ocular media and adequate pupillary dilation in the study eye, including no allergy to dilating eyedrops, to permit good quality retinal imaging.\n9. Fulfil visual acuity criteria based on ETDRS letter chart\n10. Fulfil baseline lesion size measurement, as measured by the Reading Center\n11. Evidence of disease progression as determined by the Medical Monitor following consultation with the Investigator.\n\nExclusion Criteria:\n\nSubjects must be excluded from participating in the study if they:\n\n1. Have had any intraocular surgery (including cataract surgery) or thermal laser within 90 days of the Screening Visit or planned intraocular surgery (including cataract surgery) or thermal laser during the period of the study, in the study eye.\n2. Have had any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the period of the study.\n3. Have two pathogenic or likely pathogenic variants in IRD genes (other than ABCA4) or a single pathogenic or likely pathogenic variant in autosomal dominant or X-linked IRD genes.\n4. Have a history of amblyopia in the study eye.\n5. Are unwilling to stop taking the following products at Screening and throughout the study:\n\n   1. Supplements containing vitamin A or beta-carotene, liver-based products.\n   2. Prescription oral retinoids. Topical products containing vitamin A or retinoids are not exclusionary.\n6. Have any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or intravitreal or sub-retinal or supra-choroidal injections.\n7. Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer.\n8. Have known serious allergies to the fluorescein dye that might be used to measure IOP, ocular dilating drops, topical ocular anesthetic, steroid medication, or components of the SB-007 formulation.\n9. Have any significant ocular or non-ocular disease\u002Fdisorder which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or affect the subject's ability to participate in the study.\n10. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.",{"count":55,"type":22},57,"INTERVENTIONAL",[58,59],"PHASE1","PHASE2","This Phase 1\u002F2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).\n\nThis is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.",[28,62,63],"Stargardt Macular Degeneration","Stargardt Macular Dystrophy",[65,66,31,26,67,68],"Gene Therapy","SB-007","Stargardts Disease","SGDT1","2025-04-24",{"date":71,"type":37},"2025-04-29",{"date":73,"type":37},"2025-02-11",{"date":75,"type":22},"2028-12-31",{"name":43,"class":44},5,""]