A Multi-Center, Randomized, Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of VGR-R01 in Subjects With Bietti Crystalline Dystrophy
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS
研究概览
简要总结
This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy.
This is a multicenter, randomized controlled study which will enroll 45 subjects.
详细描述
VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to assess the efficacy of VGR-R01 in subjects with Bietti crystalline dystrophy (BCD) based on the change from baseline in best corrected visual acuity (BCVA) of the study eyes.
30 subjects will be enrolled in the intervention group and will receive monocular administration of VGR-R01, and 15 subjects in the control group will not receive any intervention. After completion of the trial, the untreated eyes will receive administration of VGR-R01 in subsequent clinical trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
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);
- •Hand Motion ≤ 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.
研究组 & 干预措施
interventional group
干预措施: VGR-R01 (Drug)
结局指标
主要结局
Number of subjects with change in BCVA in the study eyes of BCD subjects as assessed by ETDRS
时间窗: Baseline up to Week 52
Change in the BCVA assessment from screening in the study eye of BCD subjects will be compared to controls
次要结局
- Change from baseline in BCVA(Baseline up to Week 52)
- Change from baseline in multi-luminance mobility test (MLMT) score of the study eye(Baseline up to Week 52)
- Changes from baseline in Microperimetry indexes(Baseline up to Week 52)
- Change from baseline in optical coherence tomography (OCT)(Baseline up to Week 52)
- Counts, frequencies and percentages of AE, SAE and other safety evaluation(Baseline up to Week 52)
- Number of subjects with the presence of immunogenicity(Baseline up to Week 52)