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Not yet recruiting NCT07586306

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

Phase I / Phase II Interventional Bietti Crystalline Dystrophy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: VGR-R01.
Who it may be relevant to
Registry conditions: Bietti Crystalline Dystrophy. Basic parameters: 18 years — 69 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

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.

Detailed description

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.

Interventions

  • Biological VGR-R01
    CYP4v2-coding gene delivered by AAV vector

Primary outcome measures

  • Incidence of adverse events and serious adverse events [Time frame: Up to Year 5]
  • Number of participants with clinically significant change from baseline in vital signs, clinically laboratory abnormalities and ophthalmic examination findings [Time frame: Up to Year 5]
Secondary outcome measures (7)
  • Change from baseline in BCVA [Time frame: Year 5]
  • Change from baseline in multi-luminance mobility test (MLMT) score [Time frame: Year 5]
  • Change from baseline in optical coherence tomography (OCT) [Time frame: Year 5]
  • Number of subjects with the presence of immunogenicity [Time frame: Year 5]
  • Number of subjects with the presence of vector shedding [Time frame: Year 5]
  • Fixation stability [Time frame: Year 5]
  • Light sensitivity [Time frame: Year 5]

Eligibility criteria

Inclusion criteria

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

Exclusion criteria

  • Have insufficient viable retinal photoreceptor cells based on investigator's decision;
  • Have current ocular or periocular infections, or endophthalmitis;
  • 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;
  • Have intraocular surgery history except cataract surgery in the study eye;
  • Have or potentially require of systemic medications that may cause eye injure;
  • Have contraindications for corticosteroids or immunosuppressant;
  • Abnormal coagulation function or other clinically significant abnormal laboratory results;
  • Have malignancies or history of malignancies;
  • History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 2 centers
  • Beijing Tongren Hospital — Beijing
  • Shanghai General Hospital (Shanghai First People's Hospital) — Shanghai

Identifiers

NCT: NCT07586306 · VGR-R01-102

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗