A Phase 1/2 Study to Assess the Safety And Efficacy Of OCU410 For Geographic Atrophy Secondary To Dry Age-Related Macular Degeneration
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Ocugen
- 入组人数
- 60
- 试验地点
- 23
- 主要终点
- Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
研究概览
简要总结
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD).
This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 60 subjects.
详细描述
Name of Sponsor/Company:
Ocugen, Inc. 11 Great Valley Parkway Malvern, PA 19355
Name of Investigational Product: OCU410
Name of Active Ingredient:
Adeno-associated viral vector 5 human RORA (AAV5-hRORA) Protocol Number: OCU410-101 Phase: 1/2 Country: US
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The following team members will be masked:
Bio-Statistician, Data Programmer, Imaging Reading Center Team, Head of Clinical Development and Medical Affairs.
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects 50 years of age or older.
- •BCVA of approximately 21 letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen equivalent).
- •Fundus autofluorescence (FAF) imaging shows:
- •Total GA area ≥2.0 and ≤20.5 mm2 (1 and 8 disk areas [DA], respectively)
- •If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 3.a
- •The entire GA lesion must be completely visualized on the macula-centered image and must be able to be imaged in its entirety, and not contiguous with any areas of peripapillary atrophy
- •Presence of any pattern of hyper-autofluorescence in the junctional zone of GA
- •Subjects who had prior treatment with an approved drug for AMD, e.g. Izerway® (Avacincaptad pegol) or Syfovre® (Pegcetacoplan injection) can be included, after a washout period of at least 3 months in study eye. Subjects can receive an approved drug for AMD in the fellow eye, if required.
排除标准
- •Previous treatment with a gene-therapy or cell therapy product
- •GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like Plaquenil maculopathy. However, benign conditions of the vitreous or peripheral retina are not exclusionary (i.e., pavingstone degeneration).
- •Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm, inability to fixate, uncontrolled glaucoma, advanced cataract, corneal abnormalities, medium haze, and other retinal pathologies.
- •Any history or current evidence of exudative ("wet") AMD including any evidence of retinal pigment epithelium rips, branch retinal artery or vein occlusion, corneal transplant, or evidence of neovascularization anywhere in the retina based on fluorescein angiogram.
研究组 & 干预措施
Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):
Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.
干预措施: OCU410 (Genetic)
Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):
Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.
干预措施: OCU410 (Genetic)
Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):
High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.
干预措施: OCU410 (Genetic)
Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm
Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.
干预措施: OCU410 (Genetic)
Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm
Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.
干预措施: OCU410 (Genetic)
Control Arm
No Intervention Control Arm: Subject will not receive any active study intervention
结局指标
主要结局
Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
时间窗: 12 months (Screening to 12 months post OCU410 administration)
The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Change in anatomy of ocular structures using Slit Lamp Biomicroscopy
时间窗: 12 months (Screening to 12 months post OCU410 administration)
We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Change in anatomy of ocular structures using Indirect ophthalmoscopy
时间窗: 12 months (Screening to 12 months post OCU410 administration)
We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Change from baseline in BCVA (Best Corrected Visual Acuity)
时间窗: 12 months (Screening to 12 months post OCU410 administration)
Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.
Change in Low Luminance Visual Acuity
时间窗: 12 months (Screening to 12 months post OCU410 administration)
Measured by letter score. A higher score represents better vision
Change in the Intraocular Pressure (mmHg)
时间窗: 12 months (Screening to 12 months post OCU410 administration)
Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).
次要结局
- Humoral and cellular immune response(12 months (Screening to 12 months post OCU410 administration))
- Shedding of viral vector(12 months (Screening to 12 months post OCU410 administration))
- Laboratory parameters including serum chemistry and hematology(12 months (Screening to 12 months post OCU410 administration))