[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"VeonGen Therapeutics GmbH\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":76},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,50],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100593127","phase-1-safety-and-preliminary-efficacy-of-vg801-in-patients-with-abca4-mutation-associated-retinal-dystrophy-stargardt-disease-100593127",false,"NCT07002398","Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)","A Single Arm, Ph1\u002F2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)","Inclusion Criteria:\n\nTo be eligible for study entry, subjects must satisfy all the following criteria:\n\n1. Written informed consent.\n2. Subjects aged ≥ 6 years.\n3. Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).\n4. Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).\n5. Poor vision in the study eye.\n\nExclusion Criteria:\n\nSubjects will be excluded from the study if one or more of the following statements are applicable to either eye:\n\n1. Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.\n2. Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.\n3. History of intraocular surgery within the previous 6 months.\n4. Previous participation in a gene therapy trial.\n5. Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.\n6. Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).","ALL","6 Years",{"count":19,"type":20},15,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.",[27,28],"Retinal Dystrophy Due to Biallelic ABCA4 Mutations","Stargardt Disease 1",[30,31,32,33,34,35,36],"ABCA4 mutation","Gene therapy","Eye disease","Stargardt disease","Retinal disease","Retinal degeneration","VG801","RECRUITING","2025-12-04",{"date":40,"type":41},"2025-12-11","ACTUAL",{"date":43,"type":41},"2024-12-23",{"date":45,"type":20},"2026-05",{"name":47,"class":48},"VeonGen Therapeutics GmbH","INDUSTRY",1,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":4,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":16,"minAge":57,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":21,"phases":60,"briefSummary":61,"conditions":62,"keywords":64,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":49},"100538522","phase-1-safety-and-tolerability-of-intravitreal-administration-of-vg901-in-patients-with-retinitis-pigmentosa-due-to-mutations-in-the-cnga1-gene-100538522","NCT06291935","Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene","A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations","Inclusion Criteria:\n\nTo be eligible for study entry, subjects must satisfy all the following criteria:\n\n1. Able to understand and willing to consent to study participation by a written informed consent\n2. Male or female ≥ 18 years of age\n3. Clinical diagnosis of RP\n4. Confirmed pathogenic, biallelic variants in the CNGA1 gene\n5. Ellipsoid zone (EZ) length of the fovea of ≥ 3000 μm in the study eye\n\nExclusion Criteria:\n\nSubjects will be excluded from the study if one or more of the following statements are applicable to either eye:\n\n1. Additional interfering ocular conditions which would impact study results (e.g., ocular opacity and advanced cataract, uveitis, amblyopia)\n2. History or presence of glaucoma\n3. Ocular surgery, intravitreal or subretinal implantation of a medical device (within 6 months of screening)\n4. Mutations known to cause inherited retinal disease other than biallelic variants in the CNGA1 gene\n5. History of ocular infection with herpes simplex virus\n6. History of ocular malignancies\n7. History of disorders of the internal retina (e.g., retinal detachment)\n8. Patients with uncontrolled diabetes (HbA1c \\> 7%)\n9. Any other retinopathy due to other diseases - including, but not limited to arterial hypertension, previous vascular retinal occlusion, trauma or acquired inflammatory diseases, contraindication to pharmacological mydriasis (e.g., history of angle block glaucoma), diabetes (diabetic retinopathy including macular oedema)\n10. Absence of visual function on the contralateral eye\n11. Any damage to the optic nerve\n12. Individuals performing any other therapy for RP within 3 months before the study, such as - but not limited to - transcorneal electrostimulation\n13. Systemic conditions (e.g., autoimmune disorders) which may affect study participation or outcome measures\n14. History of immunodeficiency or other medical conditions which may increase the risk of VG901 administration\n15. Systemic illness (e.g., hepatitis or human immunodeficiency virus \\[HIV\\] infection) or medically relevant abnormal laboratory values (3 x upper limit of normal \\[ULN\\]) in blood analysis including renal and hepatic function\n16. Current, or recent, participation in other study\u002F or administration of investigational biologic agent within 3 months of Screening; Use of any investigational agent, or systemic corticosteroids, or other immunosuppressive drug(s) within 3 months before Screening\n17. History of allergy or sensitivity to any compound used in the study\n18. Contraindications to systemic immunosuppression\n19. Subjects with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG901 administration and subjects with international normalized ratio \\> 2 or Quick \\\u003C 50% or partial thromboplastin time \\> 50 seconds, thrombocytopenia, as well as any other known coagulopathy)\n20. Subject\u002Fpartner of childbearing potential unwilling to use adequate contraception for the period between Screening and 30 days after treatment, defined as the period from Screening until 30 days after treatment (defined as administration of therapeutic to the eye)\n21. For females of childbearing potential, a positive pregnancy test at Screening or Baseline\n22. Females who are breastfeeding\n23. Previous receipt of any AAV gene therapy product\n24. Any condition which leads the investigator to believe that subject cannot comply with the protocol requirements or that may place the subject at an unacceptable risk from participating","18 Years",{"count":59,"type":20},6,[23],"The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are:\n\n* What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration?\n* Are there any early signs of efficacy on visual function?\n\nParticipants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.",[63],"Retinitis Pigmentosa",[65,66,67,32],"CNGA1 mutation","AAV2","Gene Therapy","2025-03-24",{"date":70,"type":41},"2025-03-28",{"date":72,"type":41},"2023-09-01",{"date":74,"type":20},"2026-04",{"name":47,"class":48},""]