[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Opus Genetics, Inc\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":104},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,48,78],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":31,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":5},"100645341","phase-1-phase-1b2a-dose-exploration-study-to-determine-safety-and-tolerability-of-subretinal-opgx-rdh12-1001-for-lca5-associated-inherited-retinal-degeneration-lca5-ird-100645341",false,"NCT07681778","Phase 1b\u002F2a Dose Exploration Study to Determine Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)","A Phase 1b\u002F2a Open-Label, Dose-Exploration Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-RDH12 Administered in Participants With Leber Congenital Amaurosis With Autosomal-Recessive Retinol Dehydrogenase 12 Mutations","RDH12","Inclusion Criteria:\n\n* Age ≥18 years (adult participants) or 12-17 years (adolescent participants) at the time of consent\u002Fassent.\n* Provide written informed consent and\u002For assent prior to any study procedures.\n* Willing to adhere to the clinical protocol and follow directions of the Investigator regarding post-surgery restrictions.\n* Are a good candidate for surgery, per the Investigator's judgment.\n* Have LCA with autosomal-recessive RDH12 mutation(s), confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. Historic testing, up to 15 years prior to date of consent, may be considered.\n* Clinical diagnosis of LCA with RDH12 mutation(s), in the judgment of the Investigator.\n* BCVA 20\u002F200 (1.0 logarithm of the minimum angle of resolution \\[logMAR\\]) or worse for the sentinel adult in each cohort; BCVA 20\u002F40 (0.5 logMAR) or worse for all subsequent participants in each cohort.\n\nExclusion Criteria:\n\n* Women of childbearing potential (WOCBP) who are pregnant, lactating, and\u002For unwilling to use effective contraception from Screening through 1 year after IMP administration.\n* Men who are unwilling to use effective contraception from Screening through 180 days after IMP administration.\n* Have an ocular infection, a pre-existing eye condition, or a complicating systemic disease that could preclude the planned surgery or any future ocular surgery. This includes individuals who are immunocompromised and\u002For on continuous systemic immunosuppressive therapy.\n* Have a past or current condition that may preclude participation in the study, interfere with outcome measure testing or test results, or otherwise make the potential participant unsuitable for the study.\n* Have previously received gene therapy of any kind.\n* In either eye, have undergone intraocular surgery within 90 days prior to planned IMP administration or have active inflammation at Screening resulting from prior ocular surgery.\n* Have used any investigational device or investigational drug within 90 days (or 5 half-lives of the drug, whichever is longer) prior to planned IMP administration or intend to participate in another drug or device study during the same period as the current study.\n* Have received or plan to receive a vaccination within 6 weeks prior to or 6 weeks after IMP administration. Note: For the influenza vaccine, the exclusionary period is shorter: 2 weeks prior to and 5 weeks after IMP administration (i.e., during steroid treatment).\n* Have received anticoagulant therapy within 2 weeks prior to planned IMP administration.\n* Currently use medications that are potentially neuroprotective\u002Fbeneficial or retinotoxic.\n* Are incapable of performing visual function testing (e.g., FST), with or without assistance, for reason other than poor vision.\n* Have any contraindication to a course of oral steroids, in the opinion of the Investigator.\n* Have a known history of hypersensitivity to constituents or excipients in the pharmaceutical formulation of the IMP.\n* Have a known or active infection of human immunodeficiency virus (HIV) or hepatitis B or C virus.\n* Have a known or active infection of herpes simplex virus with ocular manifestations.\n* Are an employee of the Sponsor or a relative of the Investigator or investigative site staff.","ALL","18 Years",{"count":20,"type":21},10,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This study is an early-stage clinical trial (Phase 1b\u002F2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. The treatment is delivered as a one-time injection (300 µL) into the retina (subretinal space) of the worse-seeing eye, using a method similar to approved gene therapies like Luxturna. The study is designed to evaluate safety and effectiveness at two dose levels (1E11 and 3E11 viral genomes per eye) in small groups of 5 participants. Each group begins cautiously with 2 adults (age ≥18), treated at least one month apart, followed by FDA review before allowing adolescents (ages 12-17) to participate. An independent monitoring committee (IDMC) oversees safety throughout. After 3 adolescents are treated and followed for 3 months, the committee reviews all data to decide whether to move to a higher dose. However, if the lower dose (1E11 vg\u002Feye) shows strong effectiveness in the first group, the study may expand by treating more adolescents at that same dose instead of increasing it further.",[28,29,30],"Leber Congenital Amaurosis","Leber Congenital Amaurosis (LCA)","LCA5",[32,33,34,28,30,35],"Gene therapy","IRD","LCA","OPGx-RDH12","NOT_YET_RECRUITING","2026-06-30",{"date":39,"type":40},"2026-07-02","ACTUAL",{"date":42,"type":21},"2026-09-01",{"date":44,"type":21},"2034-07",{"name":46,"class":47},"Opus Genetics, Inc","INDUSTRY",{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":54,"eligibilityCriteria":55,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":56,"targetDuration":4,"studyType":22,"phases":57,"briefSummary":58,"conditions":59,"keywords":64,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":76,"locationsCount":77},"100607184","phase-1-safety-and-tolerability-of-subretinally-injected-opgx-best1-in-patients-with-best-vitelliform-macular-dystrophy-bvmd-or-autosomal-recessive-bestrophinopathy-arb-100607184","NCT07185256","Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)","A Phase 1b\u002F2a, Open-Label, Dose-Exploration Basket Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients With Either Autosomal-Dominant BEST1 Disease (Best Vitelliform Macular Dystrophy [BVMD]) or Autosomal-Recessive Bestrophinopathy (ARB)","BIRD-1","Inclusion Criteria:\n\nIndividuals who meet all the following criteria will be eligible to participate in the study:\n\n1. Provide informed consent to study assessments.\n2. Able and willing to comply with all study assessments for the duration of the study.\n3. ≥18 years old.\n4. ETDRS BCVA measured with standard testing distances:\n\n   1. For the sentinel participant in each cohort, ≤20 letters (Snellen equivalent of 20\u002F200 \\[1.30 logMAR\\] or worse)\n   2. For subsequent participants in the same cohort, 65 to 20 letters inclusive (Snellen equivalent of 20\u002F50 \\[0.40 logMAR\\] to 20\u002F200 \\[1.30 logMAR\\]).\n5. Genetic confirmation on chromosome 11q12-q13.1 of BVMD or ARB with a BEST1 genetic test or IRD panel test including a BEST1 variant test, by a Clinical Laboratory Improvement Amendments (CLIA) or European certified laboratory. If available test result is more than 15 years old, confirmation testing will be performed at Visit 1, with results needed by Visit 3.\n6. Confirmation of one disease-causing (pathogenic or likely pathogenic, autosomal-dominant) variant in the BEST1 gene for BVMD, as listed in the IB, or two variants for ARB per American College of Medical Genetics and Genomics\u002FAssociation for Molecular Pathology (ACMG\u002FAMP) variant interpretation.\n7. BVMD: Clinical phenotype and diagnosis consistent with advanced BVMD with active subretinal fluid or vitelliform material.\n8. ARB: Clinical phenotype and diagnosis consistent with ARB.\n\nExclusion Criteria:\n\nIndividuals who meet any of the following criteria will not be eligible to participate in the study.\n\n1. Women of childbearing potential (WOCBP) who are pregnant, lactating, and\u002For unwilling to use effective contraception from Screening through 1 year after IMP administration. See Section 13.3 (Appendix 3) for contraception guidelines.\n2. Men who are unwilling to use adequate contraception from Screening through 180 days after IMP administration. See Section 13.3 (Appendix 3) for contraception guidelines.\n3. Have a pre-existing eye condition or complicating systemic disease that could preclude the planned surgery (e.g., individuals who are immunocompromised, on continuous systemic immunosuppressive treatment or anticipate a need to initiate it for non-study reasons, or unable to take the concomitant immuno-suppressive regimen necessary for IMP administration).\n4. Have a history of disease that may preclude the individual from study participation (e.g., other bestrophinopathy, such as AOFVD or ADVIRC) or that may interfere with or preclude outcome measure testing as described in the protocol.\n5. Have previously received gene therapy of any kind.\n6. Presence of active choroidal neovascularization (CNV) that, in the opinion of the Investigator, affects vision or may require treatment.\n7. Presence of subretinal fibrosis that may significantly limit improvement in visual acuity.\n8. Have an epiretinal membrane that may require surgical intervention.\n9. Have undergone tube surgery for glaucoma at any time or have glaucoma that has been unstable within the past 4 years. Note: Prior incisional surgery (e.g., trabeculectomy and iridotomy) is not exclusionary.\n10. Had intraocular surgery within 90 days prior to planned IMP administration or have active inflammation at Screening resulting from prior ocular surgery.\n11. Have used any investigational drug or device within 90 days prior to planned IMP administration or plan to participate in another drug or device study during the same period as the current study.\n12. Have received a vaccination within 6 weeks prior to planned IMP administration or plan to be vaccinated within 6 months after IMP administration.\n13. Have received anticoagulant therapy within 2 weeks prior to planned IMP administration.\n14. Have active macular neovascularization as determined by OCT-A.\n15. Are incapable of performing visual function testing for reasons other than poor vision.\n16. Have any contraindication to the concomitant steroid regiment proscribed in the protocol.\n17. Have any other condition (ocular, medical, or psychological) that would not allow the individual to complete follow-up examinations during the study and\u002For, in the opinion of the Investigator, makes it hazardous or unsuitable for the individual to participate in the study.\n18. Have a known history of hypersensitivity to constituents or excipients in the pharmaceutical formulation of the IMP.\n19. Have a known infection of human immunodeficiency virus (HIV), hepatitis B or C virus, or herpes simplex virus.\n20. Are an employee of the Sponsor or a relative of the Investigator or investigative site staff.",{"count":20,"type":21},[24,25],"The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-BEST1. The main questions it aims to answer are:\n\nEvaluate the safety and tolerability of drug OPGx-BEST1 in one eye (the treatment eye), for 5 years post-injection, in participants with BVMD or ARB.\n\nA second question it aims to answer is identification of the most appropriate dose strength of OPGx-BEST1 for clinical development.\n\nEvaluate the efficacy of single injection of OPGx-BEST1 in one eye for 5 years post-injection.\n\nWhat medical problems do participants have when taking drug OPGx-BEST1?",[60,61,62,63],"ARB","BVMD","Autosomal-Dominant Bestrophinopathy","Best Vitelliform Macular Dystrophy",[65,61,60,66,67,62],"Bestrophinopathy","OPGx-BEST1","Best vitelliform macular dystrophy","RECRUITING","2026-03-23",{"date":71,"type":40},"2026-03-24",{"date":73,"type":40},"2025-09-25",{"date":75,"type":21},"2030-08",{"name":46,"class":47},4,{"id":79,"slug":80,"hasResults":11,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":4,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":17,"minAge":85,"maxAge":4,"enrollmentInfo":86,"targetDuration":4,"studyType":22,"phases":88,"briefSummary":89,"conditions":90,"keywords":91,"overallStatus":68,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":103},"100486647","phase-1-safety-and-tolerability-subretinal-opgx-001-for-lca5-associated-inherited-retinal-degeneration-lca5-ird-and-non-interventional-arm-with-untreated-patients-100486647","NCT05616793","Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients","An Open Label, Dose Exploration, Safety and Tolerability Study of a Subretinal Injection of an OPGx-001 Gene Vector to Participants With LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) With OCncurrent Non-Interventional Follow-Up of Untreated Patients","Inclusion Criteria:\n\n1. Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures.\n2. Are willing to adhere to the clinical protocol and able to perform testing procedures.\n3. In part A participants must be 13 years of age or older at consent, for Part B, participants must be 4 years of age or older at consent with the ability to conduct the MLoMT.\n4. Carry disease-causing biallelic LCA5 gene mutations determined by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (historic testing up to 15 years from date of consent can be considered).\n5. Visual acuity: BCVA \\\u003C 20\u002F80 on the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity chart (modified for low vision participants) in the eye to be treated\n6. Show evidence of detectable photoreceptors by Spectral Domain Optical Coherence Tomography (SD-OCT)\n7. Participant is a good candidate for surgery per investigator judgement\n8. Participant agrees to follow direction of investigator regarding restrictions post-surgery (Part A only).\n\nExclusion Criteria:\n\n1. Women who are pregnant or individuals (women of childbearing potential and men) unwilling to use effective contraception for the duration of the study, including barrier methods for the first year after investigational product (IP) administration (Part A only).\n2. Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery. This includes individuals who are immunocompromised.\n3. History of intraocular surgery for either eye within 6 months prior to planned IP administration (Part A only).\n4. Have previously received gene therapy.\n5. Have used any investigational drug or device within 90 days or 5 estimated half-lives of treatment, whichever is longer or plan to participate in another study of drug or device during the study period.\n6. History of disease which may preclude the participant from participation, or which may interfere with outcome measure testing or test results.\n7. Incapable of performing visual function testing (e.g., FST testing) for reasons other than poor vision.\n8. Any absolute contraindication to a course of oral steroids.\n9. Any other condition that would not allow the potential participant to complete follow-up examinations during the study and, in the opinion of the Investigator, makes the potential participant unsuitable for the study.","4 Years",{"count":87,"type":21},22,[24,25],"The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Funding Source- FDA Office of Orphan Products Development (OOPD).",[30],[34,92,33,93,94,30],"Retinal Degeneration","adeno associated virus","gene therapy","2026-02-18",{"date":97,"type":40},"2026-02-19",{"date":99,"type":40},"2023-06-15",{"date":101,"type":21},"2028-06-15",{"name":46,"class":47},2,""]