An Open-Label Safety Study of Single-dose VGR-R01 in Patients With Bietti Crystalline Dystrophy
试验速览
- 阶段
- 早期 1 期
- 发起方
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Number of Participants with Clinically Significant Change from Baseline in Vital Signs
研究概览
简要总结
An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
详细描述
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 365(±7) days 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥18 years and <80 years of age;
- •Confirmed diagnosis of Bietti Crystalline Dystrophy;
- •Molecular diagnosis of CYP4v2 mutations (homozygotes or compound heterozygotes);
- •BCVA ≤ 20/200 in the study eye;
- •8 D <diopters< +8 D, 21 mm < axial lengths ≤ 28 mm in the study eye;
- •Normal liver function and renal function；
- •Agree to use reliable barrier contraception for 1 year after administration of VGR-R01;
- •Able to provide informed consent and comply with requirements of the study. -
排除标准
- •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;
- •Prior medications which may interfere with the interpretation of study endpoints within six months before screening, eg. anti-VEGF drugs;
- •Have or potentially require of systemic medications that may cause eye injure;
- •Live attenuated vaccines is expected to be required during the study;
- •Participation in a clinical study with an investigational drug or medical device within three months before enrollment;
- •History of allergy or sensitivity to investigational drug, medications planned for use in the study;
- •Use of anticoagulants, or after 10 days cessation of anti-platelet agents the platelet function does not recover;
- •Use of any corticosteroids, other immunosuppressive drug(s) or antipsychotic drugs (eg. antidepressant, etc.) within 3 months prior to enrollment;
- •Have contraindications for corticosteroids or immunosuppressant;
- •Have complicating systemic diseases that would preclude 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);
- •Females in lactation period;
- •Have a history of alcohol or illicit drug addiction;
- •Unable or unwilling to comply with the schedule of visits. -
研究组 & 干预措施
VGR-R01
Subretinal injection of VGR-R01
干预措施: VGR-R01 (Drug)
结局指标
主要结局
Number of Participants with Clinically Significant Change from Baseline in Vital Signs
时间窗: Baseline up to Day 365
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.
Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings
时间窗: Baseline up to Day 365
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.
Incidence of serious adverse events
时间窗: Baseline up to Day 365
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.
Number of Participants with Clinically Laboratory Abnormalities
时间窗: Baseline up to Day 365
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.
Incidence of adverse events
时间窗: Baseline up to Day 365
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.
次要结局
- Changes from Baseline in Spectral Domain Optical Coherence Tomography (SD-OCT)(Up to Day 365)
- Best-Corrected Visual Acuity (BCVA)(Up to Day 365)
- Changes from baseline in m-ERG indexes(Up to Day 365)
- Change from Baseline in NEI Visual Function Questionnaire (NEI-VFQ-25)(Up to Day 365)
- Changes from baseline in ERG indexes(Up to Day 365)
- Changes from baseline in Visual Field indexes(Up to Day 365)
- Changes from baseline in Microperimetry indexes(Up to Day 365)
- Changes from Baseline in Optical Coherence Tomography Angiography(OCT-A)(Up to Day 365)