Gene therapy SPVN20 tested in adults with rod-cone dystrophy
Tests a single eye injection of gene therapy SPVN20 in adults with advanced rod-cone dystrophy.
Plain-language title generated from the registry record
Registry title: Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy
Key facts
- Where
- Ghent, BelgiumSee the site
- Phase
- Phase 1/2
Where in Belgium
- Ghent university hospitalGhent, BelgiumTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- SparingVision
- Trial code
- NCT07807111EU 2025-520665-47-00
- Record updated
- 8 September 2026
- Original description
This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy.Read full original descriptionShow less
A Phase I/IIa Clinical Trial to Assess the Safety, Tolerability, and Efficacy of a Single Intravitreal Injection of SPVN20 Gene Therapy in Participants With Advanced Rod-Cone Dystrophy
This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy.
Inclusion: 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.
Exclusion: 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.