VG801-1, VG801-2 for ABCA4-associated retinal dystrophy

Inclusion criteria

• {"criterion_text":"- Written informed consent."}
• {"criterion_text":"- Subjects aged ≥ 6 years."}
• {"criterion_text":"- Molecular diagnosis was confirmed due to ABCA4 mutations (homozygotes or compound heterozygotes) by a CLIA (Clinical Laboratory Improvement Amendments)-certified laboratory."}
• {"criterion_text":"- Clinical diagnosis of a macular lesion phenotypically consistent with retinal disease."}
• {"criterion_text":"- Visual acuity not better than 20/60 in the study eye."}
• {"criterion_text":"- Detectable outer nuclear layer thickness in the macular region using OCT."}
• {"criterion_text":"- Study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eye drops and with sufficient fixation to permit good quality retinal imaging."}
• {"criterion_text":"- Negative pregnancy test."}
• {"criterion_text":"- Willing to avoid excessive exposure to sun light (e.g., by using a hat, ultraviolet absorbing sunglasses, and sunscreen with a minimum SPF of 30)."}

Exclusion criteria

• {"criterion_text":"- Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy."}
• {"criterion_text":"- Positive for hepatitis C or human immunodeficiency virus (HIV) infection."}
• {"criterion_text":"- Abnormal laboratory values (3  upper limit of normal [ULN]) in blood analysis including renal and hepatic function."}
• {"criterion_text":"- Subjects with increased risk of bleeding (i.e., use of anticoagulants or anti-platelet agents within 7 days before VG801 administration and subjects with international normalized ratio > 2 or Quick < 50% or partial thromboplastin time > 50 seconds, thrombocytopenia, as well as any other known coagulopathy)."}
• {"criterion_text":"- Breast-feeding woman."}
• {"criterion_text":"- Any other condition that would not allow the potential subject to complete follow-up examinations during the study and, in the opinion of the Investigator, makes the potential subject unsuitable for the study."}
• {"criterion_text":"- Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results."}
• {"criterion_text":"- History of intraocular surgery within the previous 6 months."}
• {"criterion_text":"- Previous participation in a gene therapy trial."}
• {"criterion_text":"- Previous therapy with an approved gene therapy product."}
• {"criterion_text":"- Participation in a study (investigational drug or medical device) in previous 6 months."}
• {"criterion_text":"- Incapable of performing visual function testing for reasons other than poor vision."}
• {"criterion_text":"- Any absolute contraindication to immunosuppressant regimen applied during and after administration of the IMP."}
• {"criterion_text":"- Uncontrolled diabetics (HbA1c > 7%)."}

Primary endpoints

• {"endpoint_text":"- Safety Endpoint: Number of Adverse events (ocular and systemic)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Safety Endpoint: Evaluation of the humoral immune responses against the AAV capsid","definition_or_measurement_approach":""}
• {"endpoint_text":"- Safety Endpoint: Measurement of vector in blood, tears, and saliva (vector shedding) until two negative samples have been obtained","definition_or_measurement_approach":"Vector shedding measured in blood, tears, and saliva; sampling continued until two negative samples obtained."}
• {"endpoint_text":"- Safety Endpoint: Hematology, chemistry, and urinalysis","definition_or_measurement_approach":""}
• {"endpoint_text":"- Safety Endpoint: Comprehensive ophthalmic assessments including slit lamp examination coupled with imaging including optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging.","definition_or_measurement_approach":"Comprehensive ophthalmic assessments using slit lamp exam, OCT, fundus photography, IOP monitoring, and fundus autofluorescence imaging."}
• {"endpoint_text":"- Preliminary Efficacy Endpoint: Visual Function (BCVA, Pelli Robson contrast test, OCT, fundus photography, IOP, fundus autofluorescence imaging)","definition_or_measurement_approach":"Visual function assessed by BCVA (ETDRS chart), Pelli-Robson contrast test, OCT, fundus photography, IOP, and fundus autofluorescence imaging."}
• {"endpoint_text":"- Preliminary Efficacy Endpoint: Morphological retinal measures (Fundus autofluorescence imaging , Optical coherence tomography)","definition_or_measurement_approach":"Morphological retinal measures obtained via fundus autofluorescence imaging and OCT."}
• {"endpoint_text":"- Preliminary Efficacy EndPoint: Local retinal function measures for rods and cones ( Full-field stimulus threshold testing)","definition_or_measurement_approach":"Local retinal function measured using full-field stimulus threshold testing for rods and cones."}
• {"endpoint_text":"- Preliminary Efficacy Endpoint: Retinal function measures (Goldmann kinetic visual fields, Scotopic and photopic microperimetry)","definition_or_measurement_approach":"Retinal function assessed by Goldmann kinetic visual fields and scotopic/photopic microperimetry."}
• {"endpoint_text":"- Preliminary Efficacy Endpoint: Mobility function (novel virtual reality mobility test)","definition_or_measurement_approach":"Mobility function evaluated using a novel virtual reality mobility test."}

Germany

Earliest CTIS Part Ii Submission Date

28-05-2025

Latest Decision Or Authorization Date

03-11-2025

Processing Time Days

159

Number Of Sites

2

Number Of Participants

6

Primary sponsor

Full Name

VeonGen Therapeutics GmbH

Organisation Type

Pharmaceutical company

Country Of Registered Address

Germany