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

2. september 2026 oppdatert 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.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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

Intervensjonell

Registrering (Antatt)

27

Fase

  • Fase 2
  • Fase 1

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

  • Belgia
      • Ghent, Belgia
        • Rekruttering
        • Ghent University Hospital
        • Ta kontakt med:
          • Ophthalmic Clinical Trials Unit
          • Telefonnummer: 00 32 9 332 23
          • E-post: OCTU@uzgent.be
        • Hovedetterforsker:
          • Prof. Dr. Bart Leroy
  • Frankrike
      • Paris, Frankrike, 75012
        • Rekruttering
        • CHNO XV-XX Paris - CIC 1423
        • Ta kontakt med:
        • Hovedetterforsker:
          • Dr Amine Benadji
  • Irland
      • Dublin, Irland
        • Rekruttering
        • Royal Victoria Eye and Ear Hospital
        • Ta kontakt med:
        • Hovedetterforsker:
          • Dr Emma Duignan

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

Inclusion Criteria:

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

Exclusion Criteria:

  1. 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.
  2. Participant with cortical visual impairment.
  3. Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision.
  4. Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen.
  5. Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration.
  6. Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis.
  7. 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.
  8. Active alcohol or substance abuse.
  9. Participant positive for human immunodeficiency virus (HIV) or any other systemic immunocompromising disease.
  10. Participant with active Hepatitis B or Hepatitis C.
  11. Female participant currently pregnant or breastfeeding or intending to become pregnant.
  12. Participant with clinically active ocular infection of herpetic diseases.
  13. Participant with active coronavirus disease (COVID-19) infection.
  14. Participant who received any vaccination/immunization within 28 days prior to screening and/or during screening.
  15. Participant who previously received any gene therapy product, stem cell therapy, cell-based therapy for ocular or non-ocular disease.
  16. Participant with a retinal implant at enrollment.
  17. Participant with pre-existing eye conditions that would interfere with the interpretation of study endpoints, or increase the risk of surgical complications.
  18. Participant with significant media opacity impacting evaluation of the retina or vitreous.
  19. Participant who had intraocular surgery within 90 days of study treatment administration.
  20. Participant with history of corticosteroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
  21. Participant with history of unresolved rhegmatogenous retinal detachment in the fovea.
  22. Participant with history of unresolved ocular hypotony.
  23. Participant unwilling or unable (based on the Investigator's judgment) to comply with the study protocol.
  24. 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.
  25. Participant currently taking restricted classes of medications listed in protocol
  26. Participant with ongoing cardiac or neurological disorders not managed by a restricted medication can be excluded based on the Investigator's judgement.
  27. 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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Sekvensiell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0
SPVN20
Biologisk: SPVN20 (low dose)
SPVN20
Eksperimentell: SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0
SPVN20
Biologisk: SPVN20 (medium dose)
SPVN20
Eksperimentell: SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0
SPVN20
Biologisk: SPVN20 (high dose)
SPVN20

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Safety and Tolerability
Tidsramme: 6 months
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
6 months
Ocular safety
Tidsramme: 6 months
General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
6 months

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Best Corrected Visual Acuity (BCVA)
Tidsramme: 6 months
Change in BCVA from Baseline to Month 6 after dosing with SPVN20.
6 months
Full-field stimulus threshold (FST) test
Tidsramme: 6 months
Change in FST from Baseline to Month 6 after dosing with SPVN20
6 months

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

SparingVision

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

8. oktober 2025

Primær fullføring (Antatt)

1. mars 2032

Studiet fullført (Antatt)

1. mars 2032

Datoer for studieregistrering

Først innsendt

10. august 2026

Først innsendt som oppfylte QC-kriteriene

2. september 2026

Først lagt ut (Faktiske)

8. september 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

8. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

2. september 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • SPVN20-CLIN-01
  • 2025 (U.S. NIH-stipend/kontrakt: Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
  • 2025-520665-47-00 (Ctis)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

UBESLUTTE

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Nei

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