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

NCT07807111 | Recruiting Phase 1 DMC

• 3 other identifiers: SPVN20-CLIN-012025-520665-47-002025
• Study Type: interventional
• Target: 27
• Locations: 3 countries
• Sites: 3

Brief Summary

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.

Conditions

Retinitis Pigmentosa (RP); Rod Cone Dystrophy

Keywords

Retinal Disease; Eye Disease; Retinal Degeneration; Retinal Dystrophies; Rod-Cone Dystrophy; Gene Therapy; Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (2)

• Safety and Tolerability

  Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

  6 months

• Ocular safety

  General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures

  6 months

Secondary Outcomes (2)

• Best Corrected Visual Acuity (BCVA)

  6 months

• Full-field stimulus threshold (FST) test

  6 months

Study Arms (3)

SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0

EXPERIMENTAL

SPVN20

Biological: SPVN20 (low dose)

SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0

EXPERIMENTAL

SPVN20

Biological: SPVN20 (medium dose)

SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0

EXPERIMENTAL

SPVN20

Biological: SPVN20 (high dose)

Interventions

SPVN20 (high dose) BIOLOGICAL

SPVN20

SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0

SPVN20 (low dose) BIOLOGICAL

SPVN20

SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0

SPVN20 (medium dose) BIOLOGICAL

SPVN20

SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 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.
• Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration.
• 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.
• Active alcohol or substance abuse.
• Participant positive for human immunodeficiency virus (HIV) or any other systemic immunocompromising disease.
• Participant with active Hepatitis B or Hepatitis C.
• Female participant currently pregnant or breastfeeding or intending to become pregnant.
• Participant with clinically active ocular infection of herpetic diseases.
• Participant with active coronavirus disease (COVID-19) infection.
• 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.

Sponsors & Collaborators

• SparingVision: lead

Study Sites (3)

Ghent University Hospital

Ghent, Belgium

RECRUITING

CHNO XV-XX Paris - CIC 1423

Paris, 75012, France

RECRUITING

Royal Victoria Eye And Ear Hospital

Dublin, Ireland

RECRUITING

MeSH Terms

Conditions

Retinitis Pigmentosa; Cone-Rod Dystrophies; Retinal Diseases; Eye Diseases; Retinal Degeneration; Retinal Dystrophies

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Medical Director

CONTACT

+33143462060; info@sparingvision.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Phase I/IIa open-label dose-escalation study

Study Record Dates

• First Submitted: August 10, 2026
• First Posted: September 8, 2026
• Study Start: October 8, 2025
• Primary Completion (Estimated): March 1, 2032
• Study Completion (Estimated): March 1, 2032
• Last Updated: September 8, 2026

Record last verified: 2026-09

Locations

FR (1); IE (1); BE (1)