[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"stargardt-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:stargardt-disease":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,20,0,[8,46,120,148,177,204,279,305,328,350,385,406,447,466,487,507,532,554,578,603],{"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":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100625184","phase-3-study-of-alk-001-on-the-progression-of-stargardt-disease-100625184",false,"NCT07419334","Study of ALK-001 on the Progression of Stargardt Disease","A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK 001) on the Progression of Stargardt Disease (STGD)","NORTHSTAR","Inclusion Criteria\n\n1. Male or female, 8 to 45 years of age (inclusive) on the day of screening.\n2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP.\n3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy\n4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation.\n5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.\n\nExclusion Criteria\n\n1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization.\n2. Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study.\n3. Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months.\n4. Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes.\n5. Anticipates participating in any other drug or device study within the duration of the study.","ALL","8 Years","45 Years",{"count":21,"type":22},230,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)",[28],"Stargardt Disease",[30,31,32],"Stargardt Macular Degeneration","Stargardt Macular Dystrophy","Autosomal Recessive Stargardt Disease 1 (ABCA4-related)","RECRUITING","2026-07-01",{"date":36,"type":37},"2026-07-02","ACTUAL",{"date":39,"type":22},"2026-06",{"date":41,"type":22},"2030-12",{"name":43,"class":44},"Alkeus Pharmaceuticals, Inc.","INDUSTRY",5,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":51,"acronym":52,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":55,"targetDuration":4,"studyType":23,"phases":57,"briefSummary":59,"conditions":60,"keywords":78,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":119},"100286660","stem-cell-ophthalmology-treatment-study-ii-100286660","NCT03011541","Stem Cell Ophthalmology Treatment Study II","Bone Marrow Derived Stem Cell Ophthalmology Treatment Study II","SCOTS2","Inclusion Criteria:\n\n* Have objective, documented damage to the retina or optic nerve unlikely to improve OR\n* Have objective, documented damage to the retina or optic nerve that is progressive AND have less than or equal to 20\u002F30 best corrected central visual acuity in one or both eyes AND\u002FOR an abnormal visual field in one or both eyes.\n* Be at least 3 months post-surgical treatment intended to treat any ophthalmologic disease and stable.\n* If under current medical therapy ( pharmacologic treatment) for a retinal or optic nerve disease be considered stable on that treatment and unlikely to have visual function improvement ( for example, glaucoma with intraocular pressure stable on topical medications but visual field damage ).\n* Have the potential for improvement with BMSC treatment and be at minimal risk of any potential harm from the procedure.\n* Be over the age of 18\n* Be medically stable and able to be medically cleared by their primary care physician or a licensed primary care practitioner for the procedure.\n* Medical clearance means that in the estimation of the primary care practitioner, the patient can reasonably be expected to undergo the procedure without significant medical risk to health.\n\nExclusion Criteria:\n\n* Patients who are not capable of an adequate ophthalmologic examination or evaluation to document the pathology.\n* Patients who are not capable or not willing to undergo follow up eye exams with the principle investigator or their ophthalmologist or optometrist as outlined in the protocol.\n* Patients who are not capable of providing informed consent.\n* Patients who may be at significant risk to general health or to the eyes and visual function should they undergo the procedure.","18 Years",{"count":56,"type":22},500,[58],"NA","This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.",[61,62,63,28,64,65,66,67,68,69,70,71,72,73,74,75,76,77],"Retinal Disease","Age-Related Macular Degeneration","Retinitis Pigmentosa","Optic Neuropathy","Nonarteritic Ischemic Optic Neuropathy","Optic Atrophy","Optic Nerve Disease","Glaucoma","Leber Hereditary Optic Neuropathy","Blindness","Vision Loss Night","Vision Loss Partial","Vision, Low","Retinopathy","Maculopathy","Macular Degeneration","Retina Atrophy",[79,80,81,82,83,84,85,86,87,61,76,88,89,90,91,92,93,94,95,96,63,28,97,98,99,75,67,66,64,100,101,102,103,104,105,106,107,108,69,70,109,77],"Stem Cells","Bone Marrow Derived Stem Cells","BMSC","Mesenchymal Stem Cells","MSC","Eye Disease","Ophthalmology","Ophthalmic Disease","Retina","Age Related Macular Degeneration","Myopic Macular Degeneration","Geographic Atrophy","Dry Macular Degeneration","Wet Macular Degeneration","Retinal Atrophy","Retinal Dystrophy","Hereditary Retinal Dystrophy","Malattia Leventinese","Cone Dystrophy","Rod-Cone Dystrophy","Cone-Rod Dystrophy","Ischemic Optic Neuropathy","Optic Nerve Damage","Optic Nerve Compression","Compressive Optic Neuropathy","Devics Syndrome","Ushers Syndrome","Neuromyelitis Optica","Dominant Optic Atrophy","Kjers Optic Atrophy","Vision Loss","2026-06-24",{"date":112,"type":37},"2026-06-29",{"date":114,"type":37},"2016-01",{"date":116,"type":22},"2028-07-31",{"name":118,"class":44},"MD Stem Cells",4,{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":125,"acronym":126,"eligibilityCriteria":127,"healthyVolunteers":11,"sex":17,"minAge":128,"maxAge":4,"enrollmentInfo":129,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":132,"conditions":133,"keywords":135,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":140,"lastUpdatePostDateStruct":141,"startDateStruct":142,"completionDateStruct":144,"leadSponsor":145,"locationsCount":147},"100625664","a-study-to-learn-how-stargardt-type-eye-conditions-progress-in-children-and-adults-100625664","NCT07425574","A Study to Learn How Stargardt-type Eye Conditions Progress in Children and Adults","An Observational Study of Natural Disease Progression in Participants With Macular Dystrophies Expressing a Stargardt-type Clinical Presentation","EVOLVE","Inclusion Criteria:\n\n* Participant has a documented clinical diagnosis of macular dystrophy expressing a STGD-type clinical presentation and molecular confirmation, defined as either:\n\n  * ABCA4-associated disease: presence of biallelic (pathogenic or likely pathogenic) ABCA4 variants, or one definite disease-causing ABCA4 variant together with a typical phenotype consistent with STGD.\n  * STGD-like macular dystrophy: presence of one or more pathogenic variants in a gene known to cause macular dystrophy, as appropriate for its expected inheritance mode. Note: All genetic testing should be performed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory or equivalent whenever possible. Acceptable documentation includes a copy of the genetic test report, laboratory certification statement, or clinical notes explicitly referencing CLIA certification. Predicted pathogenic variant of uncertain significance (VUS) that cannot be confirmed by standard laboratory criteria as certainly disease-causing (novel mutations) will be considered on a case by-case basis.\n* Participant has sufficiently clear ocular media and adequate pupillary dilation to allow for all imaging procedures.\n* Participant has intraocular pressure (IOP) both at screening and baseline of ≤ 21 mmHg measured by applanation tonometry. Note: Participant who is on topical IOP lowering treatment may also be included.\n* Participant has a spherical equivalent refractive error between + 8.00 D and - 10.00 D. Eyes with high myopia (\\> -10.00 D) are permitted only if there are no signs of myopic degenerative macular changes.\n* Participant has a BCVA ranging from 20\u002F500 to 20\u002F40 (equivalent to 15 to 70 ETDRS letters read at 1 meter)\n\n  * For participants in the \\> 20\u002F80 to ≤ 20\u002F40 BCVA range (moderate visual impairment \\[MVI\\]): presence of a visible definite or probable residual EZ on SD-OCT (no minimum residue size required) and a minimum central mean subfield retinal thickness on optical coherence tomography (OCT) ≥ 150 µm\n  * For participants in the ≥ 20\u002F500 to ≤ 20\u002F80 BCVA range (SVI): presence of a residual outer nuclear layer (ONL) within the macular OCT scan area and measurable within the Age-Related Eye Disease Study (AREDS) grading grid and no residual EZ or minimal thickness of the ONL required\n* Participant has evidence of retinal pigment epithelium (RPE) disease\u002Fdamage on SD-OCT (hypertransmission defects \\[HTDs\\]) and\u002For FAF imaging.\n* Participant has evidence of at least questionably decreased autofluorescence (QDAF) (incomplete retinal pigment epithelial and outer retinal atrophy \\[iRORA\\]), defined as mottled or speckled hypo-autofluorescence (AF) signal, mixed with or without hyper- AF, on FAF imaging. Note: At study onset, having patches of definitely decreased autofluorescence (DDAF) within the FAF image and\u002For patches of complete retinal pigment epithelial and outer retinal atrophy (cRORA) on SD-OCT ≥ 250 µm will not be a requirement for participation but will be acceptable for inclusion, so long as:\n\n  * DDAF\u002FcRORA areas are contained within the limits of the AREDS grading grid (≤ 12 disc areas), and physically distinct and separate from other areas of QDAF, DDAF and\u002For hyper-AF that may be present outside the AREDS grading grid, and\n  * the other BCVA and SD-OCT required inclusion criteria are also met.\n* Participant is able to comply with the protocol and willing to undertake all scheduled visits and assessments during the total study period.\n* Participant is deemed capable of performing reliably all tests required for participation.\n\nExclusion Criteria:\n\n* Participant has a known history of significant systemic disease (e.g., uncontrolled hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or immunocompromising conditions such as human immunodeficiency virus \\[HIV\\]) that could impact ocular health or confound study assessments, based on medical history or prior clinical documentation.\n* Participant has an autoimmune condition that requires treatment with immunomodulatory therapy and\u002For biologics that cause immunosuppression.\n* Participant has a known diagnosis of diabetes mellitus with a documented hemoglobin A1c (HbA1c) value ≥ 7% 3 months prior to screening and based on available medical records. If the documented HbA1c is ≥ 7% and there is no clinical history of diabetic symptoms, diabetic retinopathy, abnormal renal function (e.g., elevated creatinine), or glycosuria noted in medical records, the participant may be enrolled.\n* Participant has a known history of any systemic or metabolic condition, or physical examination finding that may significantly affect ocular health or interfere with the interpretation of study assessments.\n* Participant has a history or evidence of severe cardiac disease (e.g., New York Heart Association Functional Class III or IV), clinical evidence of unstable angina, acute coronary syndrome, myocardial infarction or revascularization within 6 months prior to screening.\n* Participant has a history or evidence of ventricular tachyarrhythmia requiring ongoing treatment.\n* Participant has a significant cardiovascular or cerebrovascular disease, including a history of stroke within 12 months prior to screening.\n* Participant has any complicating systemic disease or active malignancy. Squamous cell carcinomas will be an exclusionary criterion if lesions are recurrent and\u002For \\> 2 cm in diameter and\u002For affect the lip or ear area. Basal cell carcinomas are permitted, provided they are not located in the immediate periocular facial region. A history of any prior malignancy is allowed if the treating physician or oncologist confirms that the participant has been free of recurrence or metastasis for at least 5 years prior to screening.\n* Participant has another known or suspected molecular diagnosis of macular or retinal disease (e.g., pathogenic mutations in other genes) that could confound interpretation of study outcomes, indicate a second concomitant retinal condition or suggest a different etiology for the macular disease.\n* Participant has evidence or history of choroidal neovascularization.\n* Participant has macular atrophy due to any cause other than a genetically or clinically confirmed diagnosis of STGD or STGD-like macular dystrophies.\n* Participant has a known diagnosis of any form of uncontrolled glaucoma (for high tension glaucoma IOP \\> 25 mmHg).\n* Participant has and\u002For is receiving treatment for thyroid eye disease.\n* Participant has diabetic retinopathy in excess of mild nonproliferative diabetic retinopathy (i.e., presents with widespread retinal microaneurysms, dot-blot hemorrhages, and cotton-wool retinal spots).\n* Participant has any other disease(s) affecting the optic nerve.\n* Participant has a history of anterior or posterior uveitis and\u002For presence of intraocular inflammation (≥ trace anterior chamber cell or flare), or history of idiopathic or autoimmune-associated uveitis in either eye. Note: A single, sporadic episode of anterior uveitis without recurrences in the past 5 years and not associated with chronic conditions that increase the likelihood of recurrence may be accepted on a case-by-case basis in consultation with the sponsor's medical monitor\u002Flead.\n* Participant has corneal or lenticular opacities impeding the visualization of the fundus and\u002For the reliable performance of the visual function tests required by the protocol.\n* Participant has sufficiently narrow iridocorneal angles contraindicating pupillary dilation that have not been previously treated with preventative laser iridotomies.\n* Participant has any other ocular disorders that could interfere with or confound visual acuity and other ocular assessments, including OCT or FAF.\n* Participant has a clinically significant epiretinal membrane or evidence of clinically significant vitreomacular traction syndrome. Note: A mild enhancement of the vitreoretinal interface seen on OCT without any alteration of the foveal profile would not be considered an exclusion criterion.\n* Participant has presence of or recurrent vitreous hemorrhages. Note: A one-time episode of a traumatic vitreous hemorrhage would not result in exclusion, so long as resolved.\n* Participant has aphakia.\n* Participant has anticipated need for cataract surgery during the study period.\n* Participant has a macular hole, evident by ophthalmoscopy and\u002For by SD-OCT examinations, or history of prior macular hole surgery.\n* Participant has a history of any of the following procedures: posterior vitrectomy, retinal detachment surgery, glaucoma filtering surgery (e.g., trabeculectomy), glaucoma drainage device implantation, selective laser trabeculoplasty, full-thickness or partial-thickness corneal transplant (e.g., Descemet's stripping endothelial keratoplasty, Descemet's stripping automated endothelial keratoplasty or Descemet's membrane endothelial keratoplasty).\n* Participant has any lesion that is considered to pose a risk of retinal detachment during the study. Any preexisting lesion may be treated to allow eligibility, but treatment must be completed at least 3 months prior to screening.\n* Participant has had any prior thermal laser or photodynamic therapy treatment in the macular region, regardless of indication. Peripheral retinal thermal laser treatments for holes, lattice degeneration, and\u002For other lesions with rhegmatogenous potential are permitted within 3 months prior to screening, subject to their potential impact on a future pars plana vitrectomy surgery.\n* Participant has had any intraocular surgery within 3 months of screening.\n* Participant has a history of intraocular metallic foreign bodies.\n* Participant has received any treatment including gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or any prior intravitreal treatment for any indication in either eye that may be considered to potentially interfere with the study participation or its conduct.\n* Participant is receiving concomitant treatment with any ocular or systemic medication known to be toxic to the lens, retina or optic nerve.\n\n  * Participant is using hydroxychloroquine (Plaquenil), chloroquine, pentosan polyphosphate (Elmiron), tamoxifen, chlorpromazine, thioridazine (Mellaril), isotretinoin (Accutane) or any related retina-toxic compounds at screening.\n* Participant has received therapeutic radiation in the region of the study eye.\n* Participant has had any major surgical procedure within 1 month prior to screening or anticipates having one during the study, which may interrupt study participation.\n* Participant is taking glucagon-like peptide-1 (GLP-1) receptor agonists and is unable or unwilling to stop the medication at least 4 weeks prior to screening. A history of treatment with a GLP-1 receptor agonist is allowed.\n* Participant is taking metformin and is unable or unwilling to stop this medication at least 4 weeks prior to screening. A history of treatment with metformin is allowed.\n* Participant is participating in an interventional study of a vitamin A metabolism inhibitor, a vitamin A analog\u002Fderivative, or another investigational drug product within 3 months prior to screening. After a 3 month washout period, participants can be considered for enrollment. Participation in a noninterventional study (NIS) is allowed.\n* Participant has a known or suspected hypersensitivity to eyedrops used for IOP measurement or pupil dilation.\n* Participant has any condition that makes the participant unsuitable for study participation.","6 Years",{"count":130,"type":22},90,"OBSERVATIONAL","Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye. In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time. This leads to vision loss in the center of the eye. Side vision (peripheral vision) is mostly unaffected. Stargardt disease (STGD) is a type of macular dystrophy which is caused by 1 faulty gene (ABCA4). Vision loss most typically happens in childhood, but many people do not develop it until they are adults. As well as STGD, there are other macular dystrophies that look very similar to STGD but that are caused by many other different genes. Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies. This is because they look the same clinically and have similar symptoms. Since different genes can cause these conditions, genetic testing is the only way to be sure which specific condition a person has.\n\nIn this study, researchers want to learn if the disease progresses in a similar way in people with STGD and STGD-like macular dystrophies. People taking part in the study will continue to manage their condition, as agreed with their own doctor. People will visit their clinic every 6 months to have various standard eye tests and imaging. The information collected will include questions about people's wellbeing, general health, medication and supplements taken, and daily activities.\n\nChildren over 6 years old and adults with STGD-type macular dystrophies may take part in this study. They will be in the study for up to 24 months (2 years). The study sponsor (Astellas) will not decide how people's condition is managed. However, the sponsor will provide instructions on when people visit their clinic and what is recorded during the study. If available, medical records, clinical and imaging data from previous visits going back 24 months will also be reviewed.",[28,31,134],"Stargardt-like Macular Dystrophy",[136,137,138,139],"Macular Dystrophies","Stargardt type","ABCA4 related STGD","STGD-like macular dystrophies","2026-06-23",{"date":110,"type":37},{"date":143,"type":37},"2026-02-20",{"date":116,"type":22},{"name":146,"class":44},"Astellas Pharma Global Development, Inc.",11,{"id":149,"slug":150,"hasResults":11,"nctId":151,"briefTitle":152,"officialTitle":153,"acronym":154,"eligibilityCriteria":155,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":156,"enrollmentInfo":157,"targetDuration":4,"studyType":23,"phases":159,"briefSummary":162,"conditions":163,"keywords":164,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":168,"lastUpdatePostDateStruct":169,"startDateStruct":170,"completionDateStruct":172,"leadSponsor":174,"locationsCount":176},"100605363","phase-1-a-study-of-aavb-039-in-participants-with-stargardt-disease-stgd1-100605363","NCT07161544","A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)","An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)","CELESTE","Inclusion Criteria:\n\n* Molecular diagnosis of Stargardt disease due to ABCA4 mutation\n* Willingness to adhere to protocol per informed consent\n\nExclusion Criteria:\n\n* Unwillingness to meet the requirements of the study\n* Participation in a clinical study with another Investigation Medicinal Product\n* Previous participation in another gene or cell therapy trial\n* Any condition that would preclude subretinal surgery\n* Complicating ocular and\u002For systemic diseases","55 Years",{"count":158,"type":22},75,[160,161],"PHASE1","PHASE2","The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.",[28],[28,165,166,167],"Biallelic Mutation","Gene Mutation","ABCA4","2026-06-19",{"date":140,"type":37},{"date":171,"type":37},"2025-09-29",{"date":173,"type":22},"2032-07",{"name":175,"class":44},"AAVantgarde Bio Srl",8,{"id":178,"slug":179,"hasResults":11,"nctId":180,"briefTitle":181,"officialTitle":182,"acronym":4,"eligibilityCriteria":183,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":184,"targetDuration":4,"studyType":23,"phases":186,"briefSummary":187,"conditions":188,"keywords":190,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":194,"lastUpdatePostDateStruct":195,"startDateStruct":197,"completionDateStruct":199,"leadSponsor":201,"locationsCount":203},"100642827","phase-1-phase-1-study-of-c001-in-retinal-degeneration-100642827","NCT07594236","Phase 1 Study of C.001 in Retinal Degeneration","A Phase I Single-Center Open-label Study of C.001 in Patients With Significant Retinal Pigment Epithelium Mediated Maculopathy (RMM)","Inclusion Criteria:\n\n* Age ≥18 years\n* Diagnosis of retinal disease associated with retinal pigment epithelium degeneration (e.g., geographic atrophy or Stargardt disease)\n* Visual acuity within protocol-defined limits\n* Stable ocular condition prior to enrollment\n* Medically suitable for ophthalmic surgery and anesthesia\n* Willingness to comply with study procedures and follow-up\n\nExclusion Criteria:\n\n* Active or prior choroidal neovascularization (CNV)\n* Recent myocardial infarction or significant uncontrolled cardiovascular disease\n* Uncontrolled glaucoma\n* Significant ocular inflammatory disease\n* Recent intraocular surgery within protocol-defined timeframe\n* Active systemic infection\n* Poorly controlled diabetes (e.g., HbA1c \\>8.5%)\n* Participation in another investigational study within 3 months prior to screening",{"count":185,"type":22},15,[160],"This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium.\n\nParticipants will receive a single administration of C.001 delivered by subretinal injection. The study will evaluate three dose levels in a sequential manner.\n\nThe primary objective is to assess the safety of C.001 over a 3-month period following administration. Participants will be followed for up to 12 months to further evaluate safety and explore changes in visual function and retinal structure.",[90,28,189,88],"RPE-mediated Maculopathy",[191,192,193],"macular degeneration","macular distrophy","C.001","2026-06-10",{"date":196,"type":37},"2026-06-12",{"date":198,"type":37},"2026-04-07",{"date":200,"type":22},"2029-12-31",{"name":202,"class":44},"Cellio Therapeutics Inc",1,{"id":205,"slug":206,"hasResults":11,"nctId":207,"briefTitle":208,"officialTitle":209,"acronym":210,"eligibilityCriteria":211,"healthyVolunteers":212,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":213,"targetDuration":215,"studyType":131,"phases":4,"briefSummary":216,"conditions":217,"keywords":240,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":269,"lastUpdatePostDateStruct":270,"startDateStruct":272,"completionDateStruct":274,"leadSponsor":276,"locationsCount":203},"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":214,"type":22},20000,"20 Years","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.",[218,61,219,220,221,222,223,224,225,97,99,226,227,228,229,230,231,232,233,234,63,235,236,98,237,238,28,239],"Eye Diseases Hereditary","Achromatopsia","Bardet-Biedl Syndrome","Bassen-Kornzweig Syndrome","Batten Disease","Best Disease","Choroidal Dystrophy","Choroideremia","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 Punctata Albescens","Retinoschisis","Rod Dystrophy","Rod Monochromacy","Usher Syndrome",[241,242,243,244,245,246,247,248,249,250,251,252,253,254,255,256,257,258,259,260,261,262,263,264,265,266,267,268],"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","2026-05-18",{"date":271,"type":37},"2026-05-19",{"date":273,"type":4},"2014-06",{"date":275,"type":22},"2037-06",{"name":277,"class":278},"Foundation Fighting Blindness","OTHER",{"id":280,"slug":281,"hasResults":11,"nctId":282,"briefTitle":283,"officialTitle":284,"acronym":285,"eligibilityCriteria":286,"healthyVolunteers":11,"sex":17,"minAge":287,"maxAge":4,"enrollmentInfo":288,"targetDuration":4,"studyType":23,"phases":290,"briefSummary":291,"conditions":292,"keywords":293,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":295,"lastUpdatePostDateStruct":296,"startDateStruct":298,"completionDateStruct":300,"leadSponsor":302,"locationsCount":304},"100626765","phase-1-phase-12-open-label-dose-escalation-study-to-evaluate-safety-of-a-single-intravitreal-injection-of-rtx-021-in-patients-with-stargardt-disease-100626765","NCT07439887","Phase 1\u002F2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease","Phase 1\u002F2, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease (AURORA)","AURORA","Inclusion Criteria:\n\n* Male and female patients \\>= 16 years of age\n* Able to comply with the study visit schedule and all protocol assessments\n* Diagnosis of Stargardt Disease (genetic testing required)\n* Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria\n* Presence of retinal ganglion cells and\u002For retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center\n* Adequate organ function and general good health\n\nExclusion Criteria:\n\n* Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months\n* Concurrent participation in another interventional clinical ocular study\n* Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy\n* Pre-existing eye conditions in either eye that would preclude the planned treatment or are significant enough to interfere with the interpretation of study endpoints or procedural complications\n* Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications\n* Complicating systemic diseases including those in which the disease itself, or the treatment of the disease, can alter ocular and\u002For central nervous system function (e.g. radiation treatment of the orbit; leukemia with optic nerve involvement)\n* Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others\n* Cataract or other ocular (including refractive) surgery, intraocular and\u002For peri-ocular injection in either eye within the prior four months (i.e. 120 days) prior to screening\n* Prior vitrectomy or aphakia in the study eye\n* Known sensitivity to any component of the study treatment or contraindication to medications planned for use in the peri-procedural period (e.g. povidone-iodine to prep for intravitreal injection)\n* Known contraindication to prophylactic steroid regimen\n* Current pregnancy or breastfeeding\n* Any other condition that would not allow the patient to complete follow-up examinations during the study","16 Years",{"count":289,"type":22},18,[160,161],"A Phase 1\u002F2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.",[28],[285,294],"RTx-021","2026-04-13",{"date":297,"type":37},"2026-04-15",{"date":299,"type":37},"2026-01-28",{"date":301,"type":22},"2030-12-01",{"name":303,"class":44},"Ray Therapeutics, Inc.",3,{"id":306,"slug":307,"hasResults":11,"nctId":308,"briefTitle":309,"officialTitle":310,"acronym":311,"eligibilityCriteria":312,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":313,"targetDuration":4,"studyType":23,"phases":314,"briefSummary":315,"conditions":316,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":319,"lastUpdatePostDateStruct":320,"startDateStruct":322,"completionDateStruct":324,"leadSponsor":326,"locationsCount":203},"100613437","restoration-of-central-vision-with-prima-in-patients-with-photoreceptor-degeneration-100613437","NCT07266584","Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration","Restoration of Central Vision With the PRIMA Products in Patients With Photoreceptor Degeneration","PRIMAlia","Inclusion Criteria:\n\n1. Is 18 years or older at the date of inclusion;\n2. Has a confirmed diagnosis of inherited retinal degeneration with the macula affected in both eyes;\n3. The study eye has best corrected visual acuity of logMAR 1.2 (20\u002F320) or worse as measured by ETDRS test;\n4. Has an atrophic patch in the study eye including the fovea of at least the implant size (\\>4.5 mm2 and \\>2.4 mm in minimum diameter);\n5. Understands the constraints of the study and accepts to present for all scheduled follow-up visits;\n6. Signed the informed consent.\n\nExclusion Criteria:\n\n1. 1\\. Has cataract in the study eye (with LOCS III scale NO, NC, C or P\\>1); (these patients will need to have cataract surgery performed prior to completion of baseline testing; all other patients will get IOL replacement during the PRIMA Stim implantation);\n2. Underwent intraocular lens implantation in the study eye within the last month prior to enrolment (this corresponds to 4 weeks or 28 days) ;\n3. Has an implanted IOL in the study eye and a refraction of the study eye outside of -4D; +4D limit (this criterion is not relevant for phakic eyes)\n4. Has a highly myopic study eye (\\>26 mm AP);\n5. Has no light perception in either eye;\n6. Has a history of documented choroidal neovascularization in either eye;\n7. Has any signs of exudative AMD including exudative AMD with detachment of retinal pigment epithelium in the central visual field of the study eye;\n8. Has an implanted telescope in one eye;\n9. Has a black IOL in the study eye;\n10. Has any disease (other than study allowed diseases) or condition that affects retinal function of the study eye or the visual system (e.g., central retinal artery\u002Fvein occlusion, end-stage diabetic retinopathy, Proliferative Diabetic Retinopathy (PDR), diabetic macular oedema (DME), severe Non-Proliferative Diabetic Retinopathy (NPDR), retinal detachment, infectious or inflammatory retinal disease, severe glaucoma, optic neuropathy, etc.) ;\n11. Has any disease or condition that prevents adequate examination (including Optical Coherence Tomography (OCT)) of the study eye including but not limited to media opacities that cannot be resolved prior to implantation. Note, that this criterion is also important for the function of the implant;\n12. Has a corneal endothelial cell count of less than 1000 cells\u002Fmm² in the study eye;\n13. Suffers from nystagmus or other ocular motility disorders;\n14. Has any disease or condition that precludes the understanding or communication of the informed consent, study requirements or test protocols (e.g., deafness, severe multiple sclerosis, amyotrophic lateral sclerosis, severe neuritis, etc.);\n15. Has uncontrolled epileptic seizures;\n16. Has a known sensitivity to the contact materials of the implant (iridium oxide, silicon-carbide and titanium);\n17. Has a known allergy to anaesthetic drugs;\n18. Presents with hypotonia in the study eye (\\\u003C8 mmHg);\n19. Presents with hypertonia in the study eye (\\>23 mmHg with treatment);\n20. Has active cancer or a history of intraocular, optic nerve or brain cancer and metastasis;\n21. Is an immune-suppressed participant (e.g., due to HIV positive diagnosis, etc.);\n22. Is a known carrier of multi-resistant microorganisms;\n23. Is receiving anticoagulation therapy that cannot be adapted to allow eye surgery;\n24. Is participating in another investigational drug or device study that may interfere with the PRIMAlia study;\n25. Has a history of chronic or recurrent infection or inflammation that would preclude participation in the study;\n26. Has significant recurrent or chronic inflammations or infections. Specifically, patients with the following disorders are excluded:\n\n    * Severe chronic and consuming diseases that frequently associated with infection (e.g. Crohn disease, Whipple's disease);\n    * Active inflammation in the area of the eye (e.g. herpes of cornea and\u002For conjunctiva, recurrent blepharoconjunctivitis, hordeolum, chalazion);\n27. Has a severe psychological disorder;\n28. Does not have the mental capacity to legally sign the informed consent;\n29. Has severe renal, cardiac, hepatic, etc. organ diseases (ASA IV or worse);\n30. Has head dimensions that are incompatible with the PRIMA Companion glasses;\n31. Has too high and\u002For unrealistic expectations (e.g., believes that a benefit is guaranteed or expects normal vision after surgery).\n32. Physical constraints making use of PRIMA or rehabilitation impossible\n33. Participant who, in the opinion of the investigator, is not suitable for participation in the study",{"count":45,"type":22},[58],"The objective of this study is to evaluate the efficacy and safety of the PRIMA Products in participants with inherited retinal degeneration affecting the macula (including but not limited to Stargardt disease, and Retinitis Pigmentosa).\n\nEligible participants will be implanted with the PRIMA Stim implant. The participants will be assessed with various visual function and functional vision tests at defined timepoints throughout the clinical investigation with the PRIMA Products. The purpose of this study is to gather enough clinical data to support the clinical evaluation required for the continuous development to improve the PRIMA Products.",[28,317,318],"Retinitis Pigmentosa (RP)","Inherited Retinal Degeneration","2026-03-27",{"date":321,"type":37},"2026-04-02",{"date":323,"type":22},"2026-03-12",{"date":325,"type":22},"2031-03-01",{"name":327,"class":44},"Science Corporation",{"id":329,"slug":330,"hasResults":11,"nctId":331,"briefTitle":332,"officialTitle":332,"acronym":333,"eligibilityCriteria":334,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":335,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":337,"conditions":338,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":342,"lastUpdatePostDateStruct":343,"startDateStruct":345,"completionDateStruct":347,"leadSponsor":349,"locationsCount":203},"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",{"count":336,"type":22},25,"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.",[63,31,28,339,340,341],"Geographic Atrophy From Age-related Macular Degeneration","X-linked Retinoschisis","Retinal Dystrophies","2026-03-24",{"date":344,"type":37},"2026-03-31",{"date":346,"type":37},"2026-02-19",{"date":348,"type":22},"2027-02",{"name":303,"class":44},{"id":351,"slug":352,"hasResults":11,"nctId":353,"briefTitle":354,"officialTitle":355,"acronym":4,"eligibilityCriteria":356,"healthyVolunteers":11,"sex":17,"minAge":357,"maxAge":4,"enrollmentInfo":358,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":360,"conditions":361,"keywords":364,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":376,"lastUpdatePostDateStruct":377,"startDateStruct":379,"completionDateStruct":381,"leadSponsor":383,"locationsCount":176},"100550303","prescreening-study-to-identify-potential-stargardt-participants-for-acdn-01-clinical-trials-starpath-100550303","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.","5 Years",{"count":359,"type":22},50,"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.",[28,362,363,231],"Stargardt Disease 1","Cone Rod Dystrophy",[167,365,28,366,367,368,369,370,371,372,373,374,375],"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","2026-03-09",{"date":378,"type":37},"2026-03-11",{"date":380,"type":37},"2024-06-20",{"date":382,"type":22},"2030-08-31",{"name":384,"class":44},"Ascidian Therapeutics, Inc",{"id":386,"slug":387,"hasResults":11,"nctId":388,"briefTitle":389,"officialTitle":390,"acronym":4,"eligibilityCriteria":391,"healthyVolunteers":212,"sex":17,"minAge":54,"maxAge":156,"enrollmentInfo":392,"targetDuration":4,"studyType":23,"phases":394,"briefSummary":395,"conditions":396,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":397,"lastUpdatePostDateStruct":398,"startDateStruct":400,"completionDateStruct":402,"leadSponsor":404,"locationsCount":203},"100625048","phase-1-a-study-of-dc6001-tablet-in-healthy-chinese-adult-subjects-100625048","NCT07417566","A Study of DC6001 Tablet in Healthy Chinese Adult Subjects","A Randomized, Double-Blind, Parallel Placebo-Controlled, Single and Multiple Ascending Dose Phase I Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Characteristics of Oral DC6001 Tablet in Healthy Chinese Adult Subjects","Inclusion Criteria:\n\n1. Healthy volunteers aged 18-55 years (inclusive) at the time of signing the informed consent form, regardless of gender;\n2. Male subjects with a body weight ≥ 50.0 kg and female subjects with a body weight ≥ 45.0 kg; BMI ranging from 19.0 to 28.0 kg\u002Fm² (inclusive of critical values);\n3. Women of childbearing potential (WOCBP) or male subjects must agree to have no childbearing plans and voluntarily adopt effective contraceptive measures for 6 months from pre-screening to the last administration of the study drug, with no plans for sperm or egg donation. For WOCBP: The serum pregnancy test result of WOCBP must be negative before the first administration;\n4. No history of major diseases; physical examination, vital signs, 12-lead electrocardiogram (ECG), chest X-ray, and laboratory test results during screening are normal, or slightly exceed the normal reference range but are deemed clinically insignificant by the investigator;\n5. Subjects are able to maintain good communication with the investigator, comply with all requirements of the clinical trial, and voluntarily sign the informed consent form.\n\nExclusion Criteria:\n\n1. Subjects with clinically significant abnormalities or diseases occurring within 1 week prior to screening or currently present that require exclusion;\n2. Subjects with digestive system diseases affecting the absorption of fat-soluble vitamins occurring within 3 months prior to screening or currently present;\n3. Subjects with diseases related to vitamin A deficiency occurring within 3 months prior to screening or currently present;\n4. Subjects with acute diseases occurring from the screening phase to the first administration of the study drug that, in the investigator's judgment, may affect the study results;\n5. History of severe visual, ocular, or retinal diseases;\n6. Subjects with dysphagia;\n7. Subjects with known or suspected allergic reactions to the study drug or any of its excipients (as judged by the investigator); or subjects with clinically significant atopy or history of allergic diseases (as judged by the investigator);\n8. Subjects who have undergone surgery within 3 months prior to screening that, in the investigator's judgment, may affect drug absorption, distribution, metabolism, or excretion, or have severe surgical sequelae; or subjects planning to undergo surgery during the study;\n9. Subjects who have donated blood, lost a large amount of blood (≥ 400 mL), donated ≥ 2 units of component blood, or received blood transfusion within 3 months prior to the first administration of the study drug; or subjects planning to donate blood during the study;\n10. Subjects who have received any investigational drug or participated in any interventional clinical study within 3 months prior to the first administration of the study drug;\n11. Subjects who smoked an average of more than 5 cigarettes per day within 3 months prior to the first administration of the study drug, or cannot abstain from using any tobacco products during the study;\n12. Subjects who consumed an average of more than 14 units of alcohol per week within 3 months prior to the first administration of the study drug, or cannot abstain from using any alcohol-containing products during the study; or subjects with a positive breath alcohol test prior to study drug administration;\n13. Subjects who consumed excessive tea, coffee, and\u002For caffeinated beverages within 3 months prior to the first administration of the study drug, or cannot abstain from consuming tea, coffee, and\u002For caffeinated beverages during the study;\n14. Subjects who used any prescription drugs, over-the-counter drugs, Chinese patent medicines, Chinese herbal medicines, vitamins, or health food products within 28 days prior to screening or within 5 drug half-lives (whichever is shorter);\n15. Subjects whose 12-lead electrocardiogram (ECG) during the screening period meets any of the following criteria: 1) PR interval \\> 200 ms; 2) QTcF \\> 450 ms; 3) QRS duration \\> 110 ms; 4) QT interval \\> 500 ms; 5) Heart rate (HR) \\\u003C 50 bpm;\n16. Pregnant or lactating female subjects; or female subjects of childbearing potential (WOCBP) with a positive serum pregnancy test at any time prior to the first administration;\n17. Subjects with positive results or results exceeding the upper limit of the reference range in the eight infectious and immunological tests, which are deemed clinically significant by the investigator: hepatitis B virus (HBV) serology, hepatitis C virus (HCV) antibody, human immunodeficiency virus (HIV)-P24 antigen\u002Fantibody, and syphilis-specific antibody;\n18. Subjects with a positive urine drug screen or a history of drug abuse or drug use within 5 years prior to the study;\n19. Subjects who consumed or drank pitaya, mango, grapefruit, carambola, or foods\u002Fbeverages prepared from these fruits; or foods\u002Fbeverages containing xanthine, caffeine, or alcohol; or other special diets that may affect drug absorption, distribution, metabolism, or excretion within 72 hours prior to the first administration;\n20. Subjects with special dietary requirements, lactose intolerance, or inability to accept a unified diet;\n21. Subjects deemed unsuitable for participation in the study by the investigator.",{"count":393,"type":22},76,[160],"This study adopts a randomized, double-blind, parallel placebo-controlled dose-escalation design, consisting of two parts: Part 1 includes a single ascending dose (SAD) study plus a food effect (FE) study, and Part 2 is a multiple ascending dose (MAD) study.",[28],"2026-02-11",{"date":399,"type":37},"2026-02-18",{"date":401,"type":37},"2026-01-01",{"date":403,"type":22},"2026-12-31",{"name":405,"class":44},"Heronova Pharmaceuticals",{"id":407,"slug":408,"hasResults":11,"nctId":409,"briefTitle":410,"officialTitle":411,"acronym":412,"eligibilityCriteria":413,"healthyVolunteers":212,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":414,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":416,"conditions":417,"keywords":430,"overallStatus":437,"whyStopped":4,"lastUpdateSubmitDate":438,"lastUpdatePostDateStruct":439,"startDateStruct":441,"completionDateStruct":443,"leadSponsor":445,"locationsCount":4},"100615866","wide-field-octa-in-ocular-diseases-100615866","NCT07298174","Wide Field OCTA in Ocular Diseases","Wide Field OCTA in Ocular Diseases: a Prospective Observational Study of the Clinical Impact of Wide Field OCTA in Ocular Disease.","WOOD-2025","Inclusion Criteria:\n\n* Age \\> 18 years\n* Both genders\n* Confirmed diagnosis of one of the above diseases\n* Age-related macular degeneration\n* Diabetic retinopathy\n* Myopia\n* Pachychoroid spectrum disease\n* Inherited retinal dystrophy\n* Uveitis\n* Dry eye\n* Visual acuity of at least 1\u002F20\n* Signed informed consent for the participation to the trial.\n\nExclusion Criteria:\n\n* Media opacities\n* Any other eye or systemic condition that may irreversibly impair the results of the study\n* Surgery in the eye in the study, including cataract extraction, in the three months prior to recruitment",{"count":415,"type":22},200,"The main retinal diseases, whether or not associated with specific mutations genetic, cause progressive degeneration of vascular retinal structures and not vascular, resulting in decreased visual function. Often, such diseases affect the noblest part of the retina, called macula. Many retinal diseases can be complicated by choroidal neovascularization which causes frequent bleeding and fluid leakage that accumulates in the subretinal and intraretinal spaces. Although the investigators know many details of each disease affecting the retina, very often the correct diagnostic framework can be complicated, given the presence of morphological elements common to the different pathologies. Similarly, predicting the effect of treatment and the patient's outcome is a constant challenge for the ophthalmologists. Most of the current research has been focused on the assessment of vascular alterations localized in the macula. However, growing evidence highlight the importance of peripheral vascular changes on the outcome of retinal diseases. These changes can be detected only be wide field OCT devices.\n\nOn the other hand, ocular inflammation and hyperemia represent major assessments in anterior segment disorders, such as dry eye disease. The current grading systems of ocular inflammation, redness and hyperemia are characterized by several limitations, thus making these evaluations still mainly confined to the subjective assessment performed by the ophthalmologist. However, the new generation OCT devices may include also an anterior segment module which can reconstruct anterior segment vessels, non-invasively, using the same technology described for retinal diseases.\n\nThe main goal of the study is to evaluate the diagnostic contribution of a new generation wide field OCTA device in ocular diseases, which has recently received CE marking. In particular, the investigators will evaluate this new generation device both in retinal and anterior segments diseases, testing for common points and differences with the standard of care non-invasive diagnostic devices. Secondary outcomes include the assessment of the correlation between the patient's visual function (visual acuity) and morphological changes (standard of care imaging assessment) highlighted by the wide field OCT device, with particular attention to microstructural differences between major ocular diseases and the possible development of non-invasive biomarkers, useful for the diagnosis and follow-up of such pathologies.",[418,419,420,421,422,28,317,223,90,423,424,425,426,427,428,429],"Age - Related Macular Degeneration (AMD)","Diabetic Macular Edema","Diabetic Retinopathy","Myopia","Inherited Retinal Disease","Macular Neovascularisation","Ocular Surface Disease","Central Serous Choroidopathy","Pachychoroid Disease","Uveitis","Vitreoretinal Disease","Retinal Vein Occlusion",[431,432,433,434,435,436],"wide-field octa","multimodal retinal imaging","quantitative imaging","retinal disease","macular disease","ocular surface disease","NOT_YET_RECRUITING","2025-12-22",{"date":440,"type":37},"2025-12-30",{"date":442,"type":22},"2026-01",{"date":444,"type":22},"2028-05",{"name":446,"class":278},"IRCCS San Raffaele",{"id":448,"slug":449,"hasResults":11,"nctId":450,"briefTitle":451,"officialTitle":452,"acronym":453,"eligibilityCriteria":454,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":455,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":456,"conditions":457,"keywords":4,"overallStatus":437,"whyStopped":4,"lastUpdateSubmitDate":459,"lastUpdatePostDateStruct":460,"startDateStruct":462,"completionDateStruct":463,"leadSponsor":465,"locationsCount":203},"100613385","inherited-retinal-diseases-natural-history-and-genotype-phenotype-correlations-100613385","NCT07265895","Inherited Retinal Diseases: Natural History and Genotype-Phenotype Correlations","Inherited Retinal Diseases: Natural History and Genotype-Phenotype Correlations, Monocentric Retrospective Observational Study","IRDs-OSR","Inclusion Criteria:\n\n1. Participant completed at least one ophthalmological and retinal imaging examination at our center.\n2. Clinically diagnosed with IRD, as per familiy history, clinical signs or symptoms, retinal imaging findings.\n3. Definitive genetic diagnosis of IRD with adequate molecular test\n\nExclusion Criteria:\n\n1. Affected by other retinal or optic nerve conditions potentially affecting analyses (diabetic retinopathy, glaucoma).\n2. History of retinotoxic medications (i.e., hydroxychloroquine, pentosan polysulfate sodium, tamoxifen, ritonavir, didanosine, MEK inhibitors) intake.\n3. Unclear genetic diagnosis.\n4. Incomplete or inadequate ophthalmological and imaging tests.",{"count":415,"type":22},"Inherited Retinal Diseases (IRDs) are a heterogeneous group of genetically based degenerative retinal disorders, representing a major cause of visual impairment and blindness in working-age adults. Despite the approval of the first gene therapy for RPE65-related IRD (voretigene neparvovec) in 2017, most IRDs remain untreatable, though many gene therapies are in development. Effective trial design and therapy development require a deep understanding of disease natural history and genotype-phenotype correlations. Over 270 IRD-associated genes are known (e.g., ABCA4, USH2A, RPGR, PRPH2, BEST1), each linked to distinct phenotypes and clinical progression. This retrospective study analyzes clinical, functional, and imaging data (Optical Coherence Tomography, Fundus Autofluorescence, Microperimetry) from a large, genetically characterized IRD cohort at the IRCCS Ospedale San Raffaele up to December 31, 2025. The aims are to describe natural history, define genotype-phenotype relationships, and identify structural and functional outcome measures useful for future clinical trial endpoints, supporting personalized prognosis and trial design.",[458,317,28],"Retinal Degenerations","2025-12-04",{"date":461,"type":37},"2025-12-05",{"date":401,"type":22},{"date":464,"type":22},"2028-12-31",{"name":446,"class":278},{"id":467,"slug":468,"hasResults":11,"nctId":469,"briefTitle":470,"officialTitle":471,"acronym":4,"eligibilityCriteria":472,"healthyVolunteers":11,"sex":17,"minAge":473,"maxAge":4,"enrollmentInfo":474,"targetDuration":4,"studyType":23,"phases":475,"briefSummary":476,"conditions":477,"keywords":478,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":479,"lastUpdatePostDateStruct":480,"startDateStruct":482,"completionDateStruct":484,"leadSponsor":485,"locationsCount":486},"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":185,"type":22},[160,161],"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.",[28,363,231,362],[167,365,28,366,367,368,369,370,371,372,373,374,375],"2025-11-25",{"date":481,"type":37},"2025-12-02",{"date":483,"type":37},"2024-06-11",{"date":301,"type":22},{"name":384,"class":44},10,{"id":488,"slug":489,"hasResults":11,"nctId":490,"briefTitle":491,"officialTitle":492,"acronym":4,"eligibilityCriteria":493,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":494,"targetDuration":4,"studyType":23,"phases":495,"briefSummary":496,"conditions":497,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":499,"lastUpdatePostDateStruct":500,"startDateStruct":501,"completionDateStruct":503,"leadSponsor":505,"locationsCount":203},"100540671","phase-1-the-effects-of-disulfiram-antabuse-on-visual-acuity-in-patients-with-retinal-degeneration-100540671","NCT06319872","The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration","The Effects of Disulfiram on Visual Acuity in Patients With Retinal Degeneration","Inclusion Criteria:\n\n* All sexes, 18 years and older.\n* Participants must speak English, understand, and sign the informed consent document.\n* Stated willingness to comply with all study procedures and availability for the duration of the study.\n* In good general health as evidenced by medical history and with a clinical diagnosis of inherited retinal dystrophy or dry-AMD.\n* Best Corrected Visual Acuity (BCVA) of 20\u002F20 (with constriction or other defects of Goldmann visual field) to Light Perception in the better eye.\n* Intact inner nuclear layer, inner plexiform, and ganglion cell layer on macular SD-OCT.\n* Ability to take oral medication and be willing to adhere to the disulfiram regimen.\n* Patients must have the diagnosis of alcohol use disorder provided by an addiction specialist and be a candidate for therapeutic use of disulfiram for that condition.\n* Patients must agree to refrain from all alcohol consumption for 180 days.\n* Any female participant of childbearing potential must have a negative urine pregnancy test at screening.\n* Any female participant of childbearing potential must have (or have a partner who has) had a surgical sterilization (vasectomy, hysterectomy, or tubal ligation), be completely abstinent from intercourse or must agree to practice two acceptable methods of contraception throughout the course of the study and for at least one week after disulfiram discontinuation. 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.\n\nExclusion Criteria:\n\n* A condition that, in the opinion of the investigator, would preclude participation in the study, e.g., cardiovascular disease, hepatitis.\n* Individuals with a history of diabetes mellitus.\n* Individuals with a history of psychosis.\n* Individuals with hypothyroidism.\n* Individuals with hypersensitivity to thiuram derivatives causing rubber contact dermatitis.\n* Those on anticoagulant therapy or other medications that may be affected by disulfiram.\n* Ophthalmic conditions with independent effect upon visual function (e.g. diabetic retinopathy, glaucoma, cataract, vitreous hemorrhage, retinal detachment, active intraocular inflammation or active infectious ocular diseases, choroidal neovascularization).\n* Patients with No Light Perception (NLP) in both eyes.\n* History of major ocular surgery within the prior 6 months or major ocular surgery anticipated within the next 6 months following randomization.\n* Exam evidence of severe external ocular infection, including conjunctivitis, chalazion, or substantial blepharitis\n* Participation in an investigational trial that involves treatment with any drug within 30 days of randomization that has not received regulatory approval at the time of study entry. Note: study participants cannot receive another investigational drug while participating in this study.\n* Known allergy or hypersensitivity to any component of the study drug.\n* For women of child-bearing potential: pregnant or lactating or intending to become pregnant within the next 12 months.\n* Participants who expect to move out of the area of the clinical center during the 8 months of the study",{"count":185,"type":22},[160],"Oral disulfiram (Antabuse®) has been shown to improve image-forming vision in animal models with retinal degeneration due to its ability to decrease Retinoic Acid synthesis and consequently reduce hyperactivity in the inner retina. The investigator will aim to evaluate the impact of oral disulfiram on the vision of patients with retinal degeneration who are being treated with the drug in the management of their concurrent alcohol use disorder.",[498,341,62,63,28],"Alcohol Use Disorder","2025-09-23",{"date":171,"type":37},{"date":502,"type":37},"2025-05-19",{"date":504,"type":22},"2029-05-19",{"name":506,"class":278},"University of Rochester",{"id":508,"slug":509,"hasResults":11,"nctId":510,"briefTitle":511,"officialTitle":512,"acronym":513,"eligibilityCriteria":514,"healthyVolunteers":11,"sex":17,"minAge":473,"maxAge":515,"enrollmentInfo":516,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":517,"conditions":518,"keywords":522,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":524,"lastUpdatePostDateStruct":525,"startDateStruct":527,"completionDateStruct":529,"leadSponsor":530,"locationsCount":5},"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.","65 Years",{"count":158,"type":22},"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",[519,520,28,521],"Stargardt","Stargardt's Disease","STGD1",[513,523,521,167],"Splicebio","2025-09-18",{"date":526,"type":37},"2025-09-19",{"date":528,"type":37},"2024-03-29",{"date":348,"type":22},{"name":531,"class":44},"Splice Bio",{"id":533,"slug":534,"hasResults":11,"nctId":535,"briefTitle":536,"officialTitle":537,"acronym":538,"eligibilityCriteria":539,"healthyVolunteers":212,"sex":17,"minAge":54,"maxAge":540,"enrollmentInfo":541,"targetDuration":4,"studyType":23,"phases":543,"briefSummary":544,"conditions":545,"keywords":4,"overallStatus":437,"whyStopped":4,"lastUpdateSubmitDate":546,"lastUpdatePostDateStruct":547,"startDateStruct":548,"completionDateStruct":550,"leadSponsor":552,"locationsCount":203},"100592148","feasibility-and-tolerability-study-of-smart-contact-lens-with-healthy-subjects-and-patients-with-stargardts-disease-100592148","NCT06989658","Feasibility and Tolerability Study of Smart Contact Lens With Healthy Subjects and Patients With Stargardt's Disease","Feasibility and Tolerability Study of Wearing a Miniaturized Smart Contact Lens With Healthy Subjects and Patients With Stargardt's Disease","RE-SEE","Inclusion Criteria:\n\nPatients with Stargardt disease\n\n* Age: 18 - 70 ;\n* Juvenile form of Stargardt's disease manifested by decreased visual acuity;\n* Visual acuity greater than or equal to 20\u002F400 in binocular vision;\n* MMSE score without visual item ≥ 20\u002F25 ;\n* Eyes whose palpebral opening and ocular surface and its appendages allow the wearing of a scleral lens;\n* Sufficient knowledge of the French language.\n* Ability to give express, free and informed consent in person, after having received adequate information;\n* Ability to comply with protocol requirements;\n* Person covered by Health Insurance.\n\nHealthy volunteers:\n\n* Age: 18 - 70 years;\n* Age- and sex-matched healthy volunteers to subjects with Stargardt's disease (± 5 years);\n* MMSE score with visual items ≥ 25\u002F30 ;\n* Eyes with palpebral opening and ocular surface and appendages suitable for scleral lens wear;\n* Visual acuity of at least 10\u002F10 in binocular vision;\n* Sufficient knowledge of the French language;\n* Ability to give personal, express, written, free and informed consent after receiving adequate information ;\n* Ability to comply with protocol requirements;\n* Person covered by Health Insurance.\n\nExclusion Criteria:\n\nFor all participantsPersons referred to in articles L1121-5 to L1121-8 of the CSP (corresponding to all protected persons : pregnant women, parturients, nursing mothers, persons deprived of liberty by judicial or administrative decision, minors, and persons subject to a legal protection measure: guardianship or curatorship);\n\n* Participants with neurodegenerative diseases or any other disease that could interfere with the planned evaluations during this study;\n* Participants with ophthalmological diseases other than Stargardt's disease;\n* Participants with a history of cleft palate, corneal infection, irregular cornea (such as keratoconus) and severe dry eye syndrome as determined by the ophthalmologist;\n* Participants allergic or sensitive to methyl acrylic poly methacrylate (PMMA) or to any of the components of the Re-See lens;\n* Participants allergic to tropicamide, atropine or its derivatives\n* Participants at risk of angle-closure glaucoma;\n* Participants allergic to sodium fluorescein;\n* Suspicion of transmissible spongiform encephalopathy;\n* Medications that may cause motor, visual or cognitive disorders (neuroleptics, etc.) or that may interfere with study evaluations;\n* Participation in another clinical trial that could interfere with the present study;\n* Inability to follow instructions or read. ☞Wearing glasses or contact lenses is not a non inclusion criterion.","70 Years",{"count":542,"type":22},32,[58],"Oculometry is becoming increasingly popular in fields such as enhanced reality and healthcare, but remains limited by complex devices that are poorly adapted to the needs of users, particularly the visually impaired. This research proposes to explore a smart contact lens (SCL)-based system to overcome these limitations, particularly for people with central visual deficits such as Stargardt's disease.",[28],"2025-09-12",{"date":524,"type":37},{"date":549,"type":22},"2025-10",{"date":551,"type":22},"2028-02",{"name":553,"class":278},"Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts",{"id":555,"slug":556,"hasResults":11,"nctId":557,"briefTitle":558,"officialTitle":559,"acronym":560,"eligibilityCriteria":561,"healthyVolunteers":11,"sex":17,"minAge":473,"maxAge":515,"enrollmentInfo":562,"targetDuration":4,"studyType":23,"phases":564,"briefSummary":565,"conditions":566,"keywords":567,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":571,"lastUpdatePostDateStruct":572,"startDateStruct":574,"completionDateStruct":576,"leadSponsor":577,"locationsCount":45},"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":563,"type":22},57,[160,161],"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.",[28,30,31],[370,568,523,519,569,570],"SB-007","Stargardts Disease","SGDT1","2025-04-24",{"date":573,"type":37},"2025-04-29",{"date":575,"type":37},"2025-02-11",{"date":464,"type":22},{"name":531,"class":44},{"id":579,"slug":580,"hasResults":11,"nctId":581,"briefTitle":582,"officialTitle":583,"acronym":4,"eligibilityCriteria":584,"healthyVolunteers":212,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":585,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":587,"conditions":588,"keywords":589,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":594,"lastUpdatePostDateStruct":595,"startDateStruct":597,"completionDateStruct":599,"leadSponsor":601,"locationsCount":203},"100577990","a-prospective-observational-study-to-assess-the-reliability-and-validity-of-the-mlsdt-100577990","NCT06805474","A Prospective Observational Study to Assess the Reliability and Validity of the MLSDT","A Prospective Observational Study to Assess the Reliability and Validity of the Multi-Luminance Shape Discrimination Test (MLSDT) With 9 Objects","Inclusion Criteria:\n\n* Males and females, age ≥ 18 years\n* Able to comprehend and give informed consent.\n* Able to comply with testing and all protocol tests.\n* Eligible for 1 of 3 cohorts listed below:\n\nCohort 1: Normal-Sighted Participants with clinically normal ocular findings and BCVA range 20\u002F16 to \\\u003C20\u002F40 in each eye (ETDRS letter score 71 - 90) Cohort 2: Moderately-Sight Impaired Participants with a BCVA range of 20\u002F40 to \\\u003C20\u002F200 (ETDRS letter score of 36 - 70) and a clinical diagnosis of STGD\u002FGA Cohort 3: Severely-Sight Impaired Participants with a BCVA range of 20\u002F200 to 20\u002F800 (ETDRS letter score of 5 - 35) and a clinical diagnosis of advanced STGD\u002FGA\n\n* Clinical diagnosis of STGD for cohorts 2 and 3\n* Clinical diagnosis for GA (due to AMD) for cohorts 2 and 3\n* Participants who have had anti-VEGF therapy 2 weeks prior to enrollment have to demonstrate 3 months of vision stability and be fluid free on OCT.\n\nExclusion Criteria:\n\n* Concurrent participation in any interventional clinical trial or receipt of an investigational drug within the previous 6 months\n\n  * Presence of any condition other than STGD or GA from AMD on slit lamp exam or dilated ophthalmoscopy that impairs visual acuity or visual fields e.g., corneal opacity, visually significant cataract or visual field loss in glaucoma\n  * No intra-vitreal injection with anti-VEGF two weeks prior to the study\n  * Presence of neurological condition that impairs visual acuity\n  * Individuals who refuse or are incapable of performing the MLSDT of BCVA tests\n  * Individuals with retinal prosthesis (such as ARGUS-II)",{"count":586,"type":22},30,"The goal of this observational study is to assess the reliability and validity of the 9-object MLSDT for evaluation of participants with moderate to severe vision impairment when tested without a wearable low-vision magnifying aid (eGlasses) and then with eGlasses. These results will be compared to ETDRS testing results for the same participants without eGlasses and then with eGlasses. Two cohorts will consist of participants who have vision loss due to STGD or geographic atrophy (GA) due to age-related macular degenerations (AMD). Normally sighted participants will provide a control group.",[76,90,28],[590,591,592,593],"Multi-Luminance Shape Discrimination Test","low-vision magnifying aid (eGlasses)","best-corrected visual acuity (BCVA)","Early Treatment Diabetic Retinopathy Study ETDRS","2025-03-18",{"date":596,"type":37},"2025-03-24",{"date":598,"type":37},"2025-01-28",{"date":600,"type":22},"2025-05-27",{"name":602,"class":44},"Nanoscope Therapeutics Inc.",{"id":604,"slug":605,"hasResults":11,"nctId":606,"briefTitle":607,"officialTitle":608,"acronym":4,"eligibilityCriteria":609,"healthyVolunteers":11,"sex":17,"minAge":54,"maxAge":4,"enrollmentInfo":610,"targetDuration":4,"studyType":131,"phases":4,"briefSummary":337,"conditions":612,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":615,"lastUpdatePostDateStruct":616,"startDateStruct":618,"completionDateStruct":620,"leadSponsor":622,"locationsCount":203},"100544926","observational-study-to-assess-endpoint-operational-feasibility--measurement-properties-in-patients-with-retinal-degeneration-100544926","NCT06375239","Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration","Observational, Non-Interventional Study to Determine the Operational Feasibility and Measurement Properties of Endpoints in Patients with Retinal Degeneration","Inclusion Criteria:\n\n* Diagnosis of bilateral retinitis pigmentosa, choroideremia, Stargardt macular dystrophy, geographic atrophy from age-related macular degeneration, X-linked retinoschisis or other retinal dystrophies confirmed from previous eye examination records\n* Best-corrected visual acuity between 20\u002F70 and HM in at least one eye as tested with clinic-based visual acuity method\n* Reasonably fluent in English\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",{"count":611,"type":22},120,[63,225,31,28,613,614,341],"Geographic Atrophy from Age-related Macular Degeneration","X-lined Retinoschisis","2025-01-06",{"date":617,"type":37},"2025-01-08",{"date":619,"type":37},"2024-04-05",{"date":621,"type":22},"2026-04-30",{"name":303,"class":44}]