[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"stargardt-macular-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:stargardt-macular-dystrophy":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,46,72,108],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":28,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":34,"lastUpdatePostDateStruct":35,"startDateStruct":38,"completionDateStruct":40,"leadSponsor":42,"locationsCount":45},"100625664","a-study-to-learn-how-stargardt-type-eye-conditions-progress-in-children-and-adults-100625664",false,"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.","ALL","6 Years",{"count":20,"type":21},90,"ESTIMATED","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.",[25,26,27],"Stargardt Disease","Stargardt Macular Dystrophy","Stargardt-like Macular Dystrophy",[29,30,31,32],"Macular Dystrophies","Stargardt type","ABCA4 related STGD","STGD-like macular dystrophies","RECRUITING","2026-06-23",{"date":36,"type":37},"2026-06-24","ACTUAL",{"date":39,"type":37},"2026-02-20",{"date":41,"type":21},"2028-07-31",{"name":43,"class":44},"Astellas Pharma Global Development, Inc.","INDUSTRY",11,{"id":47,"slug":48,"hasResults":11,"nctId":49,"briefTitle":50,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":17,"minAge":53,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":56,"conditions":57,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":71},"100631590","development-and-evaluation-of-functional-visual-field-and-navigation-endpoints-in-moderate-to-profound-inherited-retinal-disease-define-ird-100631590","NCT07502664","Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)","DEFINE-IRD","Inclusion Criteria:\n\n* Diagnosis of bilateral retinitis pigmentosa (RP) or other retinal dystrophies impacting peripheral vision as confirmed from previous eye examination records\n* Best-corrected visual acuity between 20\u002F200 to HM in at least one eye.\n* Reasonably fluent in English or Spanish\n\nExclusion Criteria:\n\n* Cognitive impairment, memory loss or dementia sufficient in severity to preclude informed consent or in the opinion of the investigator would prevent satisfactory completion of some or all of the testing.\n* Any circumstance that in the opinion of the investigator, would interfere with participation in, or compliance with the study protocol\n* Current pregnancy as reported by patient","18 Years",{"count":55,"type":21},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.",[58,26,25,59,60,61],"Retinitis Pigmentosa","Geographic Atrophy From Age-related Macular Degeneration","X-linked Retinoschisis","Retinal Dystrophies","2026-03-24",{"date":64,"type":37},"2026-03-31",{"date":66,"type":37},"2026-02-19",{"date":68,"type":21},"2027-02",{"name":70,"class":44},"Ray Therapeutics, Inc.",1,{"id":73,"slug":74,"hasResults":11,"nctId":75,"briefTitle":76,"officialTitle":77,"acronym":78,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":17,"minAge":80,"maxAge":81,"enrollmentInfo":82,"targetDuration":4,"studyType":84,"phases":85,"briefSummary":88,"conditions":89,"keywords":91,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":107},"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.","12 Years","65 Years",{"count":83,"type":21},57,"INTERVENTIONAL",[86,87],"PHASE1","PHASE2","This Phase 1\u002F2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).\n\nThis is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.",[25,90,26],"Stargardt Macular Degeneration",[92,93,94,95,96,97],"Gene Therapy","SB-007","Splicebio","Stargardt","Stargardts Disease","SGDT1","2025-04-24",{"date":100,"type":37},"2025-04-29",{"date":102,"type":37},"2025-02-11",{"date":104,"type":21},"2028-12-31",{"name":106,"class":44},"Splice Bio",5,{"id":109,"slug":110,"hasResults":11,"nctId":111,"briefTitle":112,"officialTitle":113,"acronym":4,"eligibilityCriteria":114,"healthyVolunteers":11,"sex":17,"minAge":53,"maxAge":4,"enrollmentInfo":115,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":56,"conditions":117,"keywords":4,"overallStatus":33,"whyStopped":4,"lastUpdateSubmitDate":121,"lastUpdatePostDateStruct":122,"startDateStruct":124,"completionDateStruct":126,"leadSponsor":128,"locationsCount":71},"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":116,"type":21},120,[58,118,26,25,119,120,61],"Choroideremia","Geographic Atrophy from Age-related Macular Degeneration","X-lined Retinoschisis","2025-01-06",{"date":123,"type":37},"2025-01-08",{"date":125,"type":37},"2024-04-05",{"date":127,"type":21},"2026-04-30",{"name":70,"class":44}]