Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy (NYRVANA)
2 september 2026 bijgewerkt door: 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.
Studie Overzicht
Toestand
Werving
Interventie / Behandeling
Gedetailleerde beschrijving
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.
Studietype
Ingrijpend
Inschrijving (Geschat)
27
Fase
- Fase 2
- Fase 1
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studiecontact
- Naam: Medical Director
- Telefoonnummer: +33143462060
- E-mail: info@sparingvision.com
Studie Locaties
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Ghent, België
- Werving
- Ghent University Hospital
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Contact:
- Ophthalmic Clinical Trials Unit
- Telefoonnummer: 00 32 9 332 23
- E-mail: OCTU@uzgent.be
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Hoofdonderzoeker:
- Prof. Dr. Bart Leroy
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Paris, Frankrijk, 75012
- Werving
- CHNO XV-XX Paris - CIC 1423
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Contact:
- Dorothée Dagostinoz
- Telefoonnummer: 00 33 1 40 02 15 20
- E-mail: ddagostinoz@15-20.fr
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Hoofdonderzoeker:
- Dr Amine Benadji
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Dublin, Ierland
- Werving
- Royal Victoria Eye and Ear Hospital
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Contact:
- Dr Emma Duignan
- Telefoonnummer: 00 353 1 664 4600
- E-mail: Emma.duignan@rveeh.ie
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Hoofdonderzoeker:
- Dr Emma Duignan
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Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Nee
Beschrijving
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.
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Niet-gerandomiseerd
- Interventioneel model: Sequentiële toewijzing
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Experimenteel: 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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Experimenteel: 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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Experimenteel: 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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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Safety and Tolerability
Tijdsspanne: 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
Tijdsspanne: 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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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Best Corrected Visual Acuity (BCVA)
Tijdsspanne: 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
Tijdsspanne: 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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Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start (Werkelijk)
8 oktober 2025
Primaire voltooiing (Geschat)
1 maart 2032
Studie voltooiing (Geschat)
1 maart 2032
Studieregistratiedata
Eerst ingediend
10 augustus 2026
Eerst ingediend dat voldeed aan de QC-criteria
2 september 2026
Eerst geplaatst (Werkelijk)
8 september 2026
Updates van studierecords
Laatste update geplaatst (Werkelijk)
8 september 2026
Laatste update ingediend die voldeed aan QC-criteria
2 september 2026
Laatst geverifieerd
1 september 2026
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- SPVN20-CLIN-01
- 2025 (Subsidie/contract van de Amerikaanse NIH: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-520665-47-00 (Ctis)
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
ONBESLIST
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Nee
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
Nee
product vervaardigd in en geëxporteerd uit de V.S.
Nee
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