A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- Incidence of adverse events
研究概览
简要总结
A Multicenter, Open-Label, Non-Randomized, Uncontrolled 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 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.
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 69 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
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;
- •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.
结局指标
主要结局
Incidence of adverse events
时间窗: 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.
Number of Participants with Clinically Significant Change from Baseline in Vital Signs
时间窗: 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.
Number of Participants with Clinically Laboratory Abnormalities
时间窗: 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.
Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings
时间窗: 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.
Incidence of serious adverse events
时间窗: 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.
次要结局
- Changes from baseline in Mobility testing scores(Week 52)
- Changes from baseline of Mean Deviation (dB) in Visual Field (Humphery perimetry) indexes(Week 52)
- Changes from baseline of Visual Field Index (%) in Visual Field (Humphery perimetry) indexes(Week 52)
- Changes from baseline of Pattern Standard Deviation (dB) in Visual Field (Humphery perimetry) indexes(Week 52)
- Best-Corrected Visual Acuity (BCVA)(Week 52)