[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"stargardt\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:stargardt":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,41],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":5},"100611483","early-phase-1-the-safety-and-efficacy-of-zvs106e-in-the-treatment-of-irds-caused-by-biallelic-mutations-in-abca4-100611483",false,"NCT07241169","The Safety and Efficacy of ZVS106e in the Treatment of IRDs Caused by Biallelic Mutations in ABCA4","A Preliminary Clinical Study on the Safety and Efficacy of the Gene Replacement Drug ZVS106e in the Treatment of Hereditary Retinal Degeneration (IRDs) Caused by ABCA4 Biallelic Mutations","ZVS106e","Inclusion Criteria:\n\n1. Age ≥8 years old;\n2. Patients diagnosed with hereditary retinal degeneration caused by ABCA4 biallelic mutations through genetic testing, and without other ophthalmic genetic diseases;\n3. The target eye must meet the following requirements: the best corrected visual acuity is 0.5 to 2.0 LogMAR (including 0.5 and 2.0 LogMAR, equivalent to the decimal visual acuity index up to 0.3);\n4. The subject and his or her spouse agree to take effective contraceptive measures during the trial period and for at least one year after administration.\n5. Voluntarily participate in clinical trials and sign informed consent forms, and be able to complete all trial processes as required by the protocol.\n\nExclusion Criteria:\n\n1. The researchers determined that the target eye currently has or has previously had other macular lesions such as retinal schisis or epiretinal membrane. Or have other eye diseases that may hinder the surgery or interfere with the interpretation of the study endpoint;\n2. Having received drug treatment that may affect the observation of the trial within the three months prior to screening;\n3. The target eye has undergone the following intraocular surgeries: retinal repositioning and vitrectomy;\n4. There are known eye\u002Fvisual diseases, disorders or lesions that cause or are related to vision loss, or whose related treatments or therapies are known to cause or are related to vision loss;\n5. Having suffered from a viral infectious disease that may affect the efficacy and safety evaluation of the investigational drug or having received an antiviral vaccine within one month prior to enrollment;\n6. Systemic medications that are currently in use or may be required to cause eye toxicity, such as psoralen, risselinic acid or tamoxifen, etc.\n7. Known to be allergic to the drugs planned to be used in the study;\n8. Suffering from poorly drug-controlled hypertension: systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg;\n9. The following laboratory test abnormalities have clinical significance:\n\n   Liver function: Chronic liver disease, elevation of ALT or AST \\>2 times the upper limit of the normal value; Those with abnormal coagulation function (prothrombin time ≥ 3 seconds above the upper limit of the normal value, activated partial thromboplastin time ≥ 10 seconds above the upper limit of the normal value); Serum virological examination: Positive for active hepatitis B, hepatitis C virus antibody (HCV-Ab), human immunodeficiency virus antibody (HIV-Ab), or syphilis antibody;\n10. There is any past or current medical history that may affect the safety of the trial or the in vivo process of the drug, especially a history of diseases such as cardiovascular, liver, kidney, endocrine, digestive tract, lung, nervous, hematological, tumor, immune or metabolic disorders that the investigators consider to have clinical significance;\n11. Those who have participated in any clinical trials of drugs or medical devices within the three months prior to screening;\n12. Pregnant or lactating women; According to the researcher's judgment, those who are deemed unsuitable to participate in this clinical trial for other reasons","ALL","8 Years",{"count":20,"type":21},9,"ESTIMATED","INTERVENTIONAL",[24],"EARLY_PHASE1","To evaluate the preliminary safety of monocular and single subretinal injection of ZVS106e injection in the treatment of patients with hereditary retinal degeneration (ABCA4-IRDS) caused by ABCA4 biallelic mutations.",[27],"Stargardt",[15],"RECRUITING","2026-04-13",{"date":32,"type":33},"2026-04-16","ACTUAL",{"date":35,"type":33},"2025-12-29",{"date":37,"type":21},"2028-12-30",{"name":39,"class":40},"Zhongshan Ophthalmic Center, Sun Yat-sen University","OTHER",{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":17,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":54,"conditions":55,"keywords":59,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":72},"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","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.","12 Years","65 Years",{"count":52,"type":21},75,"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",[27,56,57,58],"Stargardt's Disease","Stargardt Disease","STGD1",[47,60,58,61],"Splicebio","ABCA4","2025-09-18",{"date":64,"type":33},"2025-09-19",{"date":66,"type":33},"2024-03-29",{"date":68,"type":21},"2027-02",{"name":70,"class":71},"Splice Bio","INDUSTRY",20]