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A Safety Study of Contralateral Eye Dosing of VGR-R01 in Participants With Bietti's Crystalline Dystrophy (BCD)

12. mai 2026 oppdatert av: Shanghai Vitalgen BioPharma Co., Ltd.

A Clinical Study Evaluating Subretinal Injection of VGR-R01 in the Contralateral Eye of Participants With Bietti's Crystalline Dystrophy Who Had Received VGR-R01 Administration

This is a multi-centre, single- arm, non-randomized, open-label phase 1/2 clinical trial which enables dosing of the fellow eyes of patients who received VGR-R01 administration in previous studies.

Studieoversikt

Status

Har ikke rekruttert ennå

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

VGR-R01 is a novel Adeno-associated virus (AAV) vector carrying the human Cytochrome P450 Family 4 Subfamily V Member 2 (CYP4V2) coding sequence. This study will evaluate the safety and efficacy of VGR-R01 administered in the contralateral eye (the second treated eye) of subjects enrolled in the VGR-R01-001 and VGR-R01-101 studies, along with assessments of immunogenicity and vector shedding. Additionally, the long-term safety and efficacy of VGR-R01 treatment will be continuously assessed for up to 5 years after the last dose.

Studietype

Intervensjonell

Registrering (Antatt)

15

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

  • Kina
    • Beijing Municipality
      • Beijing, Beijing Municipality, Kina
        • Beijing Tongren Hospital
        • Ta kontakt med:
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, Kina
        • Shanghai General Hospital (Shanghai First People's Hospital)
        • Ta kontakt med:

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

Key Inclusion Criteria:

  1. Participants received VGR-R01 administration in the VGR-R01-001 or VGR-R01-101 studies.
  2. Able to provide informed consent and comply with requirements of the study;
  3. Hand Motion ≤ BCVA ≤ 75 ETDRS letters in the second treated eye.

Key Exclusion Criteria:

  1. Have insufficient viable retinal photoreceptor cells based on investigator's decision;
  2. Have current ocular or periocular infections, or endophthalmitis;
  3. Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;
  4. Have intraocular surgery history except cataract surgery in the study eye;
  5. Have or potentially require of systemic medications that may cause eye injure;
  6. Have contraindications for corticosteroids or immunosuppressant;
  7. Abnormal coagulation function or other clinically significant abnormal laboratory results;
  8. Have malignancies or history of malignancies;
  9. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.

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: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: VGR-R01 group
Single-dose Subretinal Administration of VGR-R01
Biologisk: VGR-R01
CYP4v2-coding gene delivered by AAV vector

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Incidence of adverse events and serious adverse events
Tidsramme: Up to Year 5
Ocular/non-ocular adverse events are collected. The ophthalmic examination will include Best Corrected Visual Acuity (BCVA), Intraocular Pressure (IOP), slit lamp examination, angiography and Optical Coherence Tomography (OCT), etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision.
Up to Year 5
Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings
Tidsramme: Up to Year 5
Vital signs (temperature, respiratory rate, pulse rate, systolic and diastolic blood pressure) will be obtained with participant in the seated position, after having sat calmly for at least 10 minutes. Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, etc. Ophthalmic Examination will include BCVA, IOP, slit lamp examination, angiography and OCT, etc. Clinical significance of the above signs will be determined at the investigator's discretion.
Up to Year 5

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change from baseline in BCVA
Tidsramme: Year 5
BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart..
Year 5
Change from baseline in multi-luminance mobility test (MLMT) score
Tidsramme: Year 5
Subjects will navigate a standardized mobility maze under set conditions as specified times during the study. All light-levels used for testing will be rounded to one of the following specified light levels: 0.1, 1, 4, 10, 50, 125, 250, or 400 lux. The corresponding scores for the above light levels range from 7 to 0, in descending order. -1 point means failing to pass the test at the 400 lux level; the lower the score, the worse the functional vision of the participant.
Year 5
Change from baseline in optical coherence tomography (OCT)
Tidsramme: Year 5
Change from baseline in central retinal thickness (CRT) as imaged by OCT.
Year 5
Number of subjects with the presence of immunogenicity
Tidsramme: Year 5
Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product.
Year 5
Number of subjects with the presence of vector shedding
Tidsramme: Year 5
Assessed as the presence of vector in peripheral blood or collected tear.
Year 5
Fixation stability
Tidsramme: Year 5
Number of treated eyes with changes from baseline in fixation stability with MP-3 Microperimetry. Fixation stability is rated into three levels: stable fixation, relatively unstable fixation, and unstable fixation, with fixed standard reporting on the device settings.
Year 5
Light sensitivity
Tidsramme: Year 5
Changes from baseline in light sensitivity with MP-3 Microperimetry. The MP-3 has a stimulus intensity range of 0 to 34 decibels (dB).
Year 5

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Visual field index (VFI)
Tidsramme: Year 5
Assessed by Humphery static visual field testing. The VFI is a sophisticated index that range from 0% to 100%. It estimates the percentage of the visual field that the higher the better of visual function. Changes from baseline in VFI will be evaluated.
Year 5
Central threshold test
Tidsramme: Year 5
The central threshold test is a parameter in the Humphrey visual field test report, used to quantitatively assess the light sensitivity of the fovea (the area that provides the clearest central vision). It is a numerical value expressed in decibels (dB); the higher the value, the better the foveal light sensitivity.
Year 5
Change from baseline in NEI-VFQ-25 score
Tidsramme: Year 5
As assessed by the National Eye Institute Visual Function Questionnaire -25 (NEI-VFQ-25 questionnaire). NEI-VFQ-25 score ranges from 0 to 100, higher scores indicate better quality of life.
Year 5
Change from baseline in CLVQOL score
Tidsramme: Year 5
As assessed by the Chinese version of the Low Vision Quality-of-Life Questionnaire (CLVQOL questionnaire). CLVQOL score ranges from 0 to 125; higher scores indicate better quality of life.
Year 5

Samarbeidspartnere og etterforskere

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

Sponsor

Shanghai Vitalgen BioPharma Co., Ltd.

Etterforskere

  • Hovedetterforsker: Wenbin Wei, Beijing Tongren Hospital

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 (Antatt)

1. juni 2026

Primær fullføring (Antatt)

31. juli 2027

Studiet fullført (Antatt)

31. juli 2031

Datoer for studieregistrering

Først innsendt

23. april 2026

Først innsendt som oppfylte QC-kriteriene

12. mai 2026

Først lagt ut (Faktiske)

14. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

14. mai 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

12. mai 2026

Sist bekreftet

1. april 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • VGR-R01-102

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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