Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy

Ki vehet részt

• Age ≥ 18 years old at enrollment
• Participant with a genetically confirmed clinical diagnosis of advanced RCD in both eyes due to non-syndromic RCD
• Participants enrolled in the Dose-Escalation cohorts must have a BCVA meeting the study eye criteria
• Documented preservation of foveal cone cell bodies in the study eye (as shown on SD-OCT imaging)
• Participant with a history of formed vision
• Participant willing and able to provide informed consent

Ki nem vehet részt

• Participant participating in another clinical trial and receiving an investigational medicinal product (IMP) within either 5 half-lives of that IMP, or 90 days prior to the injection of SPVN20
• Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration
• Participant who received any vaccination/immunization within 28 days prior to screening and/or during screening
• Participant who previously received any gene therapy product, stem cell therapy, cell-based therapy for ocular or non-ocular disease
• Participant with a retinal implant at enrollment
• Participant who had intraocular surgery within 90 days of study treatment administration
• Participant currently taking restricted classes of medications listed in protocol
• Participant with cortical visual impairment
• Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision
• Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen
• Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis
• Participant known to be allergic to any of the delivery vehicle constituents or to any other drugs planned to be used during the clinical study
• Participant positive for human immunodeficiency virus (HIV) or any other systemic immunocompromising disease
• Participant with active Hepatitis B or Hepatitis C
• Participant with clinically active ocular infection of herpetic diseases
• Participant with active coronavirus disease (COVID-19) infection
• Participant with pre-existing eye conditions that would interfere with the interpretation of study endpoints, or increase the risk of surgical complications
• Participant with history of corticosteroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy
• Participant with history of unresolved rhegmatogenous retinal detachment in the fovea
• Participant with history of unresolved ocular hypotony
• Participant with any condition that would not allow them to complete follow-up examinations during the study and, in the opinion of the Investigator, would make them unsuitable for the study
• Participant with ongoing cardiac or neurological disorders not managed by a restricted medication can be excluded based on the Investigator's judgement
• Active alcohol or substance abuse
• Female participant currently pregnant or breastfeeding or intending to become pregnant
• Participant with significant media opacity impacting evaluation of the retina or vitreous
• Participant unwilling or unable (based on the Investigator's judgment) to comply with the study protocol
• Women of childbearing potential (WOCBP) and men and/or their partner(s) of childbearing potential who do not agree to use a highly effective contraceptive method