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NCT Number: NCT05694598

Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy

A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

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This study is active but is not currently recruiting participants.

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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

Primary location

Shanghai Vitalgen Biopharma Co.,Ltd.

Shanghai, Shanghai Municipality, China

About this study

VGR-R01 is a novel AAV vector carrying the human CYP4V2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo at least 52 weeks of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGR-R01 for a total of five years.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Able to provide informed consent and comply with requirements of the study;
  • ≥18 years and <70 years of age;
  • Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
  • BCVA ≤ 60 ETDRS letters in the study 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;
  • Unwilling or unable to have the planned follow-up;
  • 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

Genetic

CYP4v2-coding gene delivered by AAV vector

Primary outcomes

  1. Incidence of adverse events

    Time frame: Baseline up to Week 52

    An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

  2. Incidence of serious adverse events

    Time frame: Baseline up to Week 52

    A serious adverse event (SAE) is any untoward medical occurrence at any dose that resulted in death; life threatening; require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect.

  3. Number of Participants with Clinically Significant Change from Baseline in Vital Signs

    Time frame: Baseline up to Week 52

    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 5 minutes. Clinical significance of vital signs will be determined at the investigator's discretion.

  4. Number of Participants with Clinically Laboratory Abnormalities

    Time frame: Baseline up to Week 52

    Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, 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.

  5. Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings

    Time frame: Baseline up to Week 52

    Ophthalmic Examination will include BCVA, IOP, slitlamp examination, angiography and SD-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.

Secondary outcomes

  1. Best-Corrected Visual Acuity (BCVA)

    Time frame: Week 52

    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured.

  2. Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes

    Time frame: Week 52

    The VFI can range from 100% (normal visual field) to 0% (perimetrically blind field).

  3. Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes

    Time frame: Week 52

    Normal values are typically within 0dB and -2dB.

  4. Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes

    Time frame: Week 52

    A typical "normal" dB reading is around 30. The numeric dB graph should be studied next. The dBs tested by the Humphrey analyzer range between 0 and 50 dB (0 is the brightest and 50 is the dimmest).

  5. Changes from baseline in Mobility testing scores

    Time frame: Week 52

    The mobility score range is between -1 (the worst functional vision) and 6 (the best).

Other outcomes

  1. Patient reported outcome: Changes of NEI-VFQ-25

    Time frame: Week 52

    NEI-VFQ-25 questionnaire measures dimensions of self-reported vision-targeted health status that are most important to persons with eye disease. Total score ranges from 0-100, where a score of 0 represents the worst outcome and 100 represents the best outcome.

Sponsors and collaborators

Lead sponsor

Shanghai Vitalgen BioPharma Co., Ltd.

Industry

Registry information

Official study title

A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

Important dates

Study start
2023
Primary completion
2024
Study completion
2028
First posted
Jan 23, 2023
Registry last updated
Dec 24, 2025

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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