Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy (NYRVANA)
2 september 2026 uppdaterad av: SparingVision
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.
Studieöversikt
Status
Rekrytering
Betingelser
Intervention / Behandling
Detaljerad beskrivning
This is an open-label, non-randomized, multi-center, dose-escalation and dose-expansion, first-in-human, Phase I/IIa study. Participants will be followed for a total of five years after receiving a single unilateral intravitreal injection of SPVN20 in their study eye, and will be monitored by an independent Data Safety Monitoring Committee. Eligible patients will be assigned by sequential enrollment to one of the following three cohorts:
Cohort 1: low dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 2: medium dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 3: high dose of SPVN20 will be evaluated in a total of 9 participants.
Studietyp
Interventionell
Inskrivning (Beräknad)
27
Fas
- Fas 2
- Fas 1
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studiekontakt
- Namn: Medical Director
- Telefonnummer: +33143462060
- E-post: info@sparingvision.com
Studieorter
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Ghent, Belgien
- Rekrytering
- Ghent University Hospital
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Kontakt:
- Ophthalmic Clinical Trials Unit
- Telefonnummer: 00 32 9 332 23
- E-post: OCTU@uzgent.be
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Huvudutredare:
- Prof. Dr. Bart Leroy
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Paris, Frankrike, 75012
- Rekrytering
- CHNO XV-XX Paris - CIC 1423
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Kontakt:
- Dorothée Dagostinoz
- Telefonnummer: 00 33 1 40 02 15 20
- E-post: ddagostinoz@15-20.fr
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Huvudutredare:
- Dr Amine Benadji
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Dublin, Irland
- Rekrytering
- Royal Victoria Eye and Ear Hospital
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Kontakt:
- Dr Emma Duignan
- Telefonnummer: 00 353 1 664 4600
- E-post: Emma.duignan@rveeh.ie
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Huvudutredare:
- Dr Emma Duignan
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Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Nej
Beskrivning
Inclusion Criteria:
- 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 Criteria:
- 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.
- Participant with a retinal implant at enrollment.
- Participant with pre-existing eye conditions that would interfere with the interpretation of study endpoints, or increase the risk of surgical complications.
- Participant with significant media opacity impacting evaluation of the retina or vitreous.
- Participant who had intraocular surgery within 90 days of study treatment administration.
- 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 unwilling or unable (based on the Investigator's judgment) to comply with the study protocol.
- 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 currently taking restricted classes of medications listed in protocol
- Participant with ongoing cardiac or neurological disorders not managed by a restricted medication can be excluded based on the Investigator's judgement.
- 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.
Studieplan
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Icke-randomiserad
- Interventionsmodell: Sekventiell tilldelning
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
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Experimentell: SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0
SPVN20
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SPVN20
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Experimentell: SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0
SPVN20
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SPVN20
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Experimentell: SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0
SPVN20
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SPVN20
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Safety and Tolerability
Tidsram: 6 months
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Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
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6 months
|
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Ocular safety
Tidsram: 6 months
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General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
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6 months
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Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Best Corrected Visual Acuity (BCVA)
Tidsram: 6 months
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Change in BCVA from Baseline to Month 6 after dosing with SPVN20.
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6 months
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Full-field stimulus threshold (FST) test
Tidsram: 6 months
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Change in FST from Baseline to Month 6 after dosing with SPVN20
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6 months
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Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Sponsor
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart (Faktisk)
8 oktober 2025
Primärt slutförande (Beräknad)
1 mars 2032
Avslutad studie (Beräknad)
1 mars 2032
Studieregistreringsdatum
Först inskickad
10 augusti 2026
Först inskickad som uppfyllde QC-kriterierna
2 september 2026
Första postat (Faktisk)
8 september 2026
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
8 september 2026
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
2 september 2026
Senast verifierad
1 september 2026
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- SPVN20-CLIN-01
- 2025 (U.S.S. NIH-anslag/kontrakt: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-520665-47-00 (Ctis)
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