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NCT Number: NCT07586306

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

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.

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Key information

Conditions

Bietti Crystalline Dystrophy

Age range

18 year–69 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Beijing Tongren Hospital, Beijing, Beijing Municipality, China

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key 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.

Key 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.

Treatment and study plan

VGR-R01

Biological

CYP4v2-coding gene delivered by AAV vector

Primary outcomes

  1. Incidence of adverse events and serious adverse events

    Time frame: 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.

  2. 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

    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.

Secondary outcomes

  1. Change from baseline in BCVA

    Time frame: Year 5

    BCVA will be assessed with the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart..

  2. Change from baseline in multi-luminance mobility test (MLMT) score

    Time frame: 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.

  3. Change from baseline in optical coherence tomography (OCT)

    Time frame: Year 5

    Change from baseline in central retinal thickness (CRT) as imaged by OCT.

  4. Number of subjects with the presence of immunogenicity

    Time frame: Year 5

    Assessed as presence of systemic cell-mediated or humoral responses to capsid or transgene product.

  5. Number of subjects with the presence of vector shedding

    Time frame: Year 5

    Assessed as the presence of vector in peripheral blood or collected tear.

  6. Fixation stability

    Time frame: 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.

  7. Light sensitivity

    Time frame: 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).

Other outcomes

  1. Visual field index (VFI)

    Time frame: 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.

  2. Central threshold test

    Time frame: 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.

  3. Change from baseline in NEI-VFQ-25 score

    Time frame: 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.

  4. Change from baseline in CLVQOL score

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Yan Jiang

CONTACT

[email protected]

086-15802234907

Sponsors and collaborators

Lead sponsor

Shanghai Vitalgen BioPharma Co., Ltd.

Industry

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2027
Study completion
2031
First posted
May 14, 2026
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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