[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"x-linked-retinoschisis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:x-linked-retinoschisis":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,42,70,97],{"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":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":5},"100506782","phase-3-atsn-201-gene-therapy-in-rs1-associated-x-linked-retinoschisis-100506782",false,"NCT05878860","ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis","A Phase 1\u002F2\u002F3, Open-Label, Dose Escalation, Dose Expansion and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects With RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)","LIGHTHOUSE","Part A and B:\n\nInclusion Criteria:\n\n1. Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \\\u003C 18 years for Cohort 5.\n2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1.\n3. Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20\u002F200 to 20\u002F40).\n4. Presence of foveal schisis and \u002For parafoveal\u002Fperifoveal schisis in the study eye on SD-OCT per the Principal Investigator.\n\nExclusion Criteria:\n\n1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).\n2. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening or any intraocular surgery anticipated in the study eye during the first 12 months of the study.\n3. Treatment in a prior ocular gene or cell therapy study.\n\nPart C:\n\nInclusion Criteria:\n\nNote: For patients ineligible for bilateral dosing based on the ocular exclusion criteria, the same eye must meet all ocular inclusion criteria, but none of the ocular exclusion criteria, to be eligible for unilateral dosing.\n\nGeneral All of the following criteria must be met for unilateral or bilateral dosing.\n\n1. Age ≥ 6 years.\n2. Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or likely pathogenic mutations in RS1.\n\n   Ocular At least 1 eye must meet all of the following criteria for both unilateral and bilateral dosing.\n3. BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20\u002F200 to 20\u002F40).\n4. Presence of foveal schisis on SD-OCT.\n\nExclusion Criteria:\n\nGeneral None of the following criteria can be met for unilateral or bilateral dosing.\n\n1. Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening.\n2. Treatment in a prior ocular gene or cell therapy study.\n3. Absence of macular schisis.\n4. BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20\u002F32).\n5. Pre-existing eye conditions that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).\n6. Any intraocular surgery (including laser treatment) within 6 months prior to Screening or any intraocular surgery anticipated during the first 12 months of the study.","ALL","6 Years",{"count":20,"type":21},97,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).",[27],"X-linked Retinoschisis",[29],"XLRS, RS1","RECRUITING","2026-04-24",{"date":33,"type":34},"2026-04-30","ACTUAL",{"date":36,"type":34},"2023-08-22",{"date":38,"type":21},"2033-04",{"name":40,"class":41},"Atsena Therapeutics Inc.","INDUSTRY",{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":17,"minAge":49,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":52,"phases":4,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":69},"100631590","development-and-evaluation-of-functional-visual-field-and-navigation-endpoints-in-moderate-to-profound-inherited-retinal-disease-define-ird-100631590","NCT07502664","Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)","DEFINE-IRD","Inclusion Criteria:\n\n* Diagnosis of bilateral retinitis pigmentosa (RP) or other retinal dystrophies impacting peripheral vision as confirmed from previous eye examination records\n* Best-corrected visual acuity between 20\u002F200 to HM in at least one eye.\n* Reasonably fluent in English or Spanish\n\nExclusion Criteria:\n\n* Cognitive impairment, memory loss or dementia sufficient in severity to preclude informed consent or in the opinion of the investigator would prevent satisfactory completion of some or all of the testing.\n* Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance with the study protocol\n* Current pregnancy as reported by patient","18 Years",{"count":51,"type":21},25,"OBSERVATIONAL","The Vision Research and Assessment Institute (VRAI) was established with the purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision.\n\nThe mission of the VRAI is to enable the highest quality, standardized efficacy testing of patients with visual impairment.\n\nThe VRAI facilitates the development and refinement of existing endpoints specifically for testing patients with Low Vision.",[55,56,57,58,27,59],"Retinitis Pigmentosa","Stargardt Macular Dystrophy","Stargardt Disease","Geographic Atrophy From Age-related Macular Degeneration","Retinal Dystrophies","2026-03-24",{"date":62,"type":34},"2026-03-31",{"date":64,"type":34},"2026-02-19",{"date":66,"type":21},"2027-02",{"name":68,"class":41},"Ray Therapeutics, Inc.",1,{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":4,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":49,"enrollmentInfo":79,"targetDuration":4,"studyType":22,"phases":81,"briefSummary":83,"conditions":84,"keywords":86,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":87,"lastUpdatePostDateStruct":88,"startDateStruct":90,"completionDateStruct":92,"leadSponsor":94,"locationsCount":69},"100542671","early-phase-1-safety-and-efficacy-of-a-single-subretinal-injection-of-jwk002-gene-therapy-in-subjects-with-x-linked-retinoschisisxlrs-100542671","NCT06345898","Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)","Clinical Study on the Safety and Efficacy of JWK002 Injection Administered as a Single Subretinal Injection in Subjects With X-linked Retinoschisis","Inclusion Criteria:\n\n1. Fully understand the purpose and requirements of the trial, voluntarily participate in the clinical trial and sign the informed consent, and be able to cooperate with the testing required in various studies according to the requirements of the program;\n2. Male, ≥5 years old and \\\u003C 18 years old (subject to the date of signing the informed consent);\n3. Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;\n4. Best corrected visual acuity (BCVA) of the study eye ≤63 letters (equivalent to Snellen 20\u002F63); Best corrected visual acuity (BCVA) ≥34 letters (equivalent to Snellen 20\u002F200).\n\nExclusion Criteria:\n\n1. Carbonic anhydrase inhibitor treatment within 3 months;\n2. The presence of other conditions in the study eye that may cause vision loss (e.g., atrophy of the optic nerve, advanced glaucoma, uveitis, the presence of vesicular splitting chambers or vesicular retinal detachment that may affect the subretinal injection operator);\n3. Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity;\n4. There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint;\n5. Patients who had undergone internal eye surgery within 6 months;\n6. Patients with AAV8 neutralizing antibody titer ≥1:1000;\n7. Have previously received any gene therapy or cell therapy;\n8. fertile subjects are unwilling to use contraception;\n9. The presence of any of the following: an active infection that the investigator believes may affect the patient's participation in the study or affect the study results that requires systematic treatment; Hepatitis B surface antigen positive, hepatitis B virus deoxyribonucleic acid (HBV DNA) copy number \\> ULN; Hepatitis C virus (HCV) antibody positive and HCV-RNA copy number \\>ULN; Treponema pallidum antibody positive; Positive for human immunodeficiency virus (HIV) antibodies;\n10. Malignant tumors (basal cell or squamous cell skin cancer) were diagnosed within 5 years prior to screening;\n11. has or has had a systemic immune system disease;\n12. Laboratory values considered to be clinically significant were abnormal: alanine and\u002For aspartate transaminase \\>2.5×ULN, total bilirubin \\>1.5×ULN, serum creatinine \\>1.5×ULN, prothrombin time ≥1.5×ULN, activated partial thromboplastin time ≥1.5×ULN; (13) have a severe allergy or a known allergy to the drugs used in the treatment or examination in the study protocol, including the investigational drug allergy;\n\n(14) Other situations that the investigator considers inappropriate for participation in this study.","MALE","5 Years",{"count":80,"type":21},12,[82],"EARLY_PHASE1","This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002.",[85],"X Linked Retinoschisis",[85],"2025-02-04",{"date":89,"type":34},"2025-02-07",{"date":91,"type":34},"2023-11-17",{"date":93,"type":21},"2033-11-30",{"name":95,"class":96},"West China Hospital","OTHER",{"id":98,"slug":99,"hasResults":11,"nctId":100,"briefTitle":101,"officialTitle":102,"acronym":4,"eligibilityCriteria":103,"healthyVolunteers":11,"sex":77,"minAge":18,"maxAge":4,"enrollmentInfo":104,"targetDuration":4,"studyType":22,"phases":105,"briefSummary":107,"conditions":108,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":69},"100501876","safety-and-efficacy-study-of-lx103-treatment-of-x-linked-retinoschisis-xlrs-100501876","NCT05814952","Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)","An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)","Inclusion Criteria:\n\n1. Willing to sign the informed consent, and willing to attend follow-up visits.\n2. Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene.\n3. The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.\n\nExclusion Criteria:\n\n1. Any eye with disease that would interfere with the fundus examinations.\n2. The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.\n3. The study eye has undergone intraocular surgery within six months prior to enrollment.\n4. Participant has uncontrolled hypertension or diabetes.",{"count":80,"type":21},[106],"NA","The goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.",[27],"2023-04-04",{"date":111,"type":34},"2023-04-18",{"date":113,"type":34},"2022-12-13",{"date":115,"type":21},"2028-08",{"name":117,"class":96},"Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine"]