[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"usher-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:usher-syndrome":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,42,125,157,194],{"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":22,"briefSummary":24,"conditions":25,"keywords":28,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":4},"100615278","phase-3-24-month-trial-of-npi-001-for-the-preservation-of-photoreceptors-in-retinitis-pigmentosa-associated-with-usher-syndrome-100615278",false,"NCT07290530","24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome","A 24-Month, Randomized, Double-Masked, Placebo-Controlled Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome","Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an informed consent form (ICF) and to adhere to the study protocol.\n2. Diagnosed with Usher syndrome.\n3. EZ zone width ≥ 500 microns, which includes the fovea in each eye at Visit 1 (Screening).\n4. All edges of the EZ area in both eyes can be visualized at Visit 1 (Screening).\n5. Have at least 20 detectable points on the MAIA grid in at least one eye at the Screening and Baseline visits (same eye for both visits).\n\n5\\. On stable dose of medications associated with other conditions for at least one month.\n\n6\\. Both female participants of childbearing potential and male participants able to father children must have (or have a partner who has) had a bilateral oophorectomy, hysterectomy or bilateral salpingectomy; must abstain from intercourse; or must agree to practice 2 acceptable methods of contraception throughout the course of the study and 4 weeks after the last visit. Acceptable methods of contraception include hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring), intrauterine device, barrier methods (diaphragm, condom) with spermicide, tubal ligation, and vasectomy.\n\nExclusion Criteria:\n\n1. Concurrent retinal pathologies that result in vision loss or inability to fixate, including but not limited to, choroideremia, retinal vein occlusion, and neovascular age-related macular degeneration.\n2. Intraocular surgery within the last two months or capsulotomy within the last month.\n3. Current or history of uveitis, Coat's disease, diabetic retinopathy, glaucoma, herpes simplex of the eye, or currently has a cataract that prevents visualization of the posterior pole.\n4. Likely to require cataract surgery within the next 12 months.\n5. Unstable fixation during microperimetry in either eye at either Screening or Baseline visits.\n6. Use of any other investigational new drug, or participation in another clinical trial within 12 weeks before the start of study treatment.\n7. Use of N-acetylcysteine containing products in the previous 30 days prior to the baseline visit or unwilling to refrain from such supplements for the duration of the study.\n8. Chronic liver or kidney disease, cystic fibrosis, severe asthma, or chronic obstructive pulmonary disease (COPD), history of thrombocytopenia not due to a reversible cause, or other blood dyscrasia.\n9. Suspected liver dysfunction determined by having alanine aminotransferase (ALT), aspartate aminotransferase (AST), or bilirubin values \\> 1.5 X the upper limit of normal (ULN) at screening.\n10. Platelet or hemoglobin values \\\u003C 100 at screening.\n11. History of known sensitivity to N-acetylcysteine or similar thiol compounds or any ingredients of NPI-001.\n12. History of hypersensitivity to any medication or food resulting in systemic symptoms.\n13. History of cancer (other than non-melanoma skin cancer) diagnosed or requiring treatment within the past 2 years.\n14. Pregnant women or women planning to become pregnant in the next 25 months or men with partners planning to become pregnant in the next 25 months.\n15. Lactating women who are breast-feeding.\n16. Potential participant lives in the same household as a current participant in this study.\n17. Subjects who are willing or unable to refrain from donation of blood from 30 days prior to Screening, plasma from 2 weeks prior to Screening, or platelets from 6 weeks prior to Screening until 56 days following the last study drug dose administered.\n18. Inability to provide blood samples, including difficulty with venous access.\n19. Any reason, in the opinion of the Principal Investigator, the participant should not participate.","ALL","18 Years",{"count":19,"type":20},80,"ESTIMATED","INTERVENTIONAL",[23],"PHASE3","The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001. The main questions it aims to answer are:\n\nDoes NPI-001 slow down the loss of photoreceptors? What medical problems do participants have when taking NPI-001? Researchers will compare NPI-001 to a placebo (a look-alike substance that contains no drug) to see if NPI-001 works to preserve vision.\n\nParticipants will:\n\nTake NPI-001 or a placebo twice a day, every day for 24 months Visit the clinic 9 times for checkups and tests",[26,27],"Retinitis Pigmentosa (RP)","Usher Syndrome",[29],"N-acetylcysteine amide","NOT_YET_RECRUITING","2026-06-28",{"date":33,"type":34},"2026-07-01","ACTUAL",{"date":36,"type":20},"2026-12",{"date":38,"type":20},"2029-07",{"name":40,"class":41},"Nacuity Pharmaceuticals, Inc.","INDUSTRY",{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":48,"eligibilityCriteria":49,"healthyVolunteers":50,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":51,"targetDuration":53,"studyType":54,"phases":4,"briefSummary":55,"conditions":56,"keywords":84,"overallStatus":113,"whyStopped":4,"lastUpdateSubmitDate":114,"lastUpdatePostDateStruct":115,"startDateStruct":117,"completionDateStruct":119,"leadSponsor":121,"locationsCount":124},"100242565","inherited-retinal-degenerative-disease-registry-100242565","NCT02435940","Inherited Retinal Degenerative Disease Registry","Foundation Fighting Blindness My Retina Tracker Registry","MRTR","Inclusion Criteria:\n\n* Diagnosed with an inherited retinal degenerative disease OR\n\nExclusion Criteria:\n\n* Glaucoma only\n* Diabetic retinopathy only\n* Non-retinal disease\n* Not heritable retinal disease",true,{"count":52,"type":20},20000,"20 Years","OBSERVATIONAL","The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation. It is a patient-initiated registry accessible via a secure on-line portal at www.MyRetinaTracker.org. Affected individuals who register are guided to create a profile that captures their perspective on their retinal disease and its progress; family history; genetic testing results; preventive measures; general health and interest in participation in research studies. The participants may also choose to ask their clinician to add clinical measurements and results at each clinical visit. Participants are urged to update the information regularly to create longitudinal records of their disease, from their own perspective, and their clinical progress. The overall goals of the Registry are: to better understand the diversity within the inherited retinal degenerative diseases; to understand the prevalence of the different diseases and gene variants; to assist in the establishment of genotype-phenotype relationships; to help understand the natural history of the diseases; to help accelerate research and development of clinical trials for treatments; and to provide a tool to investigators that can assist with recruitment for research studies and clinical trials.",[57,58,59,60,61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,80,81,82,83,27],"Eye Diseases Hereditary","Retinal Disease","Achromatopsia","Bardet-Biedl Syndrome","Bassen-Kornzweig Syndrome","Batten Disease","Best Disease","Choroidal Dystrophy","Choroideremia","Cone Dystrophy","Cone-Rod Dystrophy","Congenital Stationary Night Blindness","Enhanced S-Cone Syndrome","Fundus Albipunctatus","Goldmann-Favre Syndrome","Gyrate Atrophy","Juvenile Macular Degeneration","Kearns-Sayre Syndrome","Leber Congenital Amaurosis","Refsum Syndrome","Retinitis Pigmentosa","Retinitis Punctata Albescens","Retinoschisis","Rod-Cone Dystrophy","Rod Dystrophy","Rod Monochromacy","Stargardt Disease",[85,86,87,88,89,90,91,92,93,94,95,96,97,98,99,100,101,102,103,104,105,106,107,108,109,110,111,112],"inherited retinal degenerative disease","retinitis pigmentosa","Usher","Leber","Bardet-Biedl","Batten","Best","cone dystrophy","cone-rod dystrophy","choroideremia","congenital night blindness","enhanced s-cone","cone monochromacy","Goldmann-Favre","Kearns-Sayre","Refsum","retinoschisis","rod-cone dystrophy","rod dystrophy","rod monochromacy","Sorsby pseudoinflammatory dystrophy","stargardt","achromatopsia","juvenile inherited macular degeneration","cone dichromacy","cone trichromacy","Charcot-Marie-Tooth","albipunctate dystrophy","RECRUITING","2026-05-18",{"date":116,"type":34},"2026-05-19",{"date":118,"type":4},"2014-06",{"date":120,"type":20},"2037-06",{"name":122,"class":123},"Foundation Fighting Blindness","OTHER",1,{"id":126,"slug":127,"hasResults":11,"nctId":128,"briefTitle":129,"officialTitle":130,"acronym":131,"eligibilityCriteria":132,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":133,"targetDuration":4,"studyType":54,"phases":4,"briefSummary":135,"conditions":136,"keywords":138,"overallStatus":113,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":155,"locationsCount":124},"100635150","observational-study-to-investigate-the-short-term-effects-of-transcorneal-electrical-stimulation-on-visual-performance-100635150","NCT07548944","Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual Performance","Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation (TES) on Visual Performance in Patients With Retinitis Pigmentosa and Similar Retinal Diseases","TES-STEP","Inclusion Criteria:\n\n* Adult patients (≥ 18 years) with clinically diagnosed retinitis pigmentosa (RP) or other similar degenerative retinal disease\n* Current treatment with TES using the OkuStim® System within the intended purpose\n* Subjective perception of short-term effects after TES treatment\n* Ability and willingness to give informed consent\n\nExclusion Criteria:\n\n* Cognitive, psychological, or linguistic limitations that prevent informed consent or proper study participation\n* Presence of other eye diseases (e.g., cataract, glaucoma, macular degeneration) that, in the opinion of the investigator, compromise or confound study assessments\n* Simultaneous participation in other clinical studies that could influence the results of the study\n* Severe comorbidities that could compromise patient safety or study conduct",{"count":134,"type":20},30,"Retinitis pigmentosa and similar degenerative diseases of the retina lead to progressive loss of vision. TES therapy with the CE-marked OkuStim® System is a treatment approved in the EU for slowing the progression of the disease. Patients increasingly report short-term subjective improvements in vision, which have not yet been systematically investigated. This exploratory study is conducted to determine whether these subjective short-term effects can be measured, and therefore also be quantified, by objective tests.",[26,27,137],"Cone Rod Dystrophy",[139,140,141,142,143,144,145,146,147],"Transcorneal Electrical Stimulation","TES","OkuStim","Okuvision","Retinitis pigmentosa","Eye Diseases","Retinal Dystrophies","Retinal Degeneration","Retinal Diseases","2026-04-30",{"date":150,"type":34},"2026-05-01",{"date":152,"type":34},"2026-04-20",{"date":154,"type":20},"2027-03",{"name":156,"class":41},"Okuvision GmbH",{"id":158,"slug":159,"hasResults":11,"nctId":160,"briefTitle":161,"officialTitle":162,"acronym":163,"eligibilityCriteria":164,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":165,"targetDuration":4,"studyType":21,"phases":167,"briefSummary":170,"conditions":171,"keywords":176,"overallStatus":113,"whyStopped":4,"lastUpdateSubmitDate":184,"lastUpdatePostDateStruct":185,"startDateStruct":187,"completionDateStruct":189,"leadSponsor":191,"locationsCount":193},"100576757","phase-1-a-study-to-investigate-the-safety-of-opct-001-in-adults-who-have-primary-photoreceptor-disease-clarico-100576757","NCT06789445","A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)","A Phase 1\u002F2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease","CLARICO","Key Inclusion Criteria:\n\n* Confirmed genetic diagnosis of primary photoreceptor (PR) disease\n* Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.\n* Retinal structure examination in the study eye demonstrating regions suitable for cell administration.\n\nKey Exclusion Criteria:\n\n* Clinically relevant, active ocular inflammation or infection\n* Glaucoma or other significant optic neuropathy\n* Diabetic macular edema or diabetic retinopathy\n* Clinically significant cystoid macular edema\n* In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia\n* Ocular surgery ≤3 months before Screening\n* Monocular vision (ie, no light perception in the fellow eye)\n* Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.\n* Any current and active infection (bacterial\u002Fviral\u002Ffungal) that could put the participant at risk from immunosuppression\n* History of any cell therapy, gene therapy, or retinal implant at any time\n* Previously received a bone marrow or solid organ transplant",{"count":166,"type":20},54,[168,169],"PHASE1","PHASE2","Study OpCT-001-101 is a Phase 1\u002F2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.",[172,26,27,173,80,174,146,175,67],"Primary Photoreceptor Disease","Inherited Retinal Disease (IRD)","Rod-Cone Disease","Cone-Rod Disease (C-RD)",[163,177,178,179,27,77,180,181,182,183],"Photoreceptor cells","Cell Therapy","Cellular Therapy","Inherited retinal disease (IRD)","Primary Photoreceptor disease (PPD)","Rod-Con Disease (R-CD)","Cone-Rose disease (C-RD)","2026-04-07",{"date":186,"type":34},"2026-04-13",{"date":188,"type":34},"2025-03-10",{"date":190,"type":20},"2030-10",{"name":192,"class":41},"BlueRock Therapeutics",4,{"id":195,"slug":196,"hasResults":11,"nctId":197,"briefTitle":198,"officialTitle":199,"acronym":200,"eligibilityCriteria":201,"healthyVolunteers":11,"sex":16,"minAge":202,"maxAge":203,"enrollmentInfo":204,"targetDuration":206,"studyType":54,"phases":4,"briefSummary":207,"conditions":208,"keywords":209,"overallStatus":113,"whyStopped":4,"lastUpdateSubmitDate":215,"lastUpdatePostDateStruct":216,"startDateStruct":218,"completionDateStruct":220,"leadSponsor":222,"locationsCount":124},"100614380","natural-history-of-photoreceptor-degeneration-in-ush1b-clinical-parameters-and-validation-of-functional-vision-tests-in-myo7a-100614380","NCT07278843","Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7A","Natural History of Photoreceptor Degeneration Related to USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7A","MYO7A","Inclusion Criteria:\n\n* Be at least 3 years old;\n* Have a clinical diagnosis of USH1 in both eyes, meaning subjects with congenital profound deafness, vestibular dysfunction, and rod dystrophy, carrying biallelic class 4 or 5 variants in the MYO7A gene;\n* Be affiliated with or beneficiary of a social security system (according to article L1121-8-1 of the French Public Health Code);\n\nFor participants in the MOST-VR mobility test and VR-ViSA visual search test (Streetlab), additional criteria apply:\n\n* Sufficient knowledge of spoken and signed French to ensure understanding of tasks and instructions;\n* Have a cochlear implant allowing comprehension of auditory instructions for the virtual reality mobility test and a MMSE score ≥ 20\u002F25;\n* Age between 18 and 75 years.\n\nExclusion Criteria:\n\n* Unable to participate in all study visits;\n* Expected to enter an experimental treatment trial at any time during this study;\n* Presence of ocular conditions that may affect eye status other than retinitis pigmentosa (e.g., history of retinal detachment, glaucoma, vein occlusion, diabetic retinopathy, etc.);\n* Participation in the previous gene replacement trial (USHSTAT, NCT01505062);\n* Pregnant, delivering, or breastfeeding women (according to article L1121-5 of the French Public Health Code);\n* Persons deprived of liberty by judicial or administrative decision (article L1121-6 of the French Public Health Code);\n* Adults under legal protection measures or unable to provide consent (article L1121-8 of the French Public Health Code).\n\nFor participants in the MOST-VR mobility and VR-ViSA visual search tests, the following non-inclusion criteria apply:\n\n* MMSE score without visual items ≤ 20\u002F25;\n* Physical or cognitive impairment that could interfere with mobility;\n* Medication that may cause motor, visual, or cognitive disorders (e.g., APS, neuroleptics) or interfere with study assessments.","3 Years","75 Years",{"count":205,"type":20},60,"48 Months","Inherited retinal diseases (IRDs) are a group of degenerative disorders that cause progressive vision loss. Retinitis pigmentosa (RP) is the most common form, with a global prevalence of approximately 1 in 4,500. About 20-30% of these cases are syndromic, most notably Usher syndrome (USH), which combines hearing loss with visual impairment. Usher syndrome type 1 (USH1), the most severe form, presents at birth with profound sensorineural hearing loss, vestibular areflexia, and early-onset retinal degeneration. Biallelic mutations in the MYO7A gene, which define the USH1B subtype, account for 70% of USH1 cases. There is currently no treatment available for this serious condition. The objective of the study is to characterize the natural history of retinal degeneration in USH1B patients and to validate functional vision tests using virtual reality and patient-reported outcome questionnaires.",[27],[210,211,212,213,214],"Retina","Genetic mutation","Inherited disease","Retinis pigmentosa","Usher syndrome","2026-02-09",{"date":217,"type":34},"2026-02-10",{"date":219,"type":34},"2025-10-13",{"date":221,"type":20},"2032-09",{"name":223,"class":123},"Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts"]