NCT05984927进行中（未招募）1 期
A Phase 1/2a Open-label Study to Evaluate Safety, Tolerability and Preliminary Efficacy of NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration
Elisigen, Inc.14 个研究点 分布在 2 个国家目标入组 20 人开始时间: 2023年9月8日最近更新:
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 20
- 试验地点
- 14
- 主要终点
- Adverse Events
研究概览
简要总结
This study will evaluate the safety, tolerability, and preliminary efficacy of NG101 AAV gene therapy administered by subretinal injections into a single selected eye as a single selected dose for patients with wet age-related macular degeneration (wAMD).
详细描述
This is a Phase 1/2a, multicenter, open-label, dose escalation study to evaluate the safety, tolerability, and preliminary efficacy of NG101 AAV gene therapy, administered by subretinal injection in patients with active wAMD symptoms. The study will be conducted at approximately 5 sites in Canada and the USA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 50 Years 至 89 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects ≥ 50 and ≤ 89 years of age with a diagnosis of active subfoveal CNV secondary to wAMD in the Study Eye
- •BCVA between 20/40 and 20/400 in the Study Eye, with BCVA decrement primarily attributable to wAMD
- •Administration of at least 3 anti-VEGF (vascular endothelial growth factor) injections in the study eye in the past 6 months for clinically active wAMD, the most recent of which was within 1 month prior to Screening Day -14 (Visit 1).
- •Must be pseudo phakic (status post cataract surgery) in the Study Eye
- •Female and Male reproductive status: Female subjects must be either: (1) of non-childbearing potential; or (2) of childbearing potential and using an acceptable method of birth control with a negative pregnancy test. Male subjects agree to refrain from sperm donations and practice contraception to avoid any pregnancy for 3 months after administration of NG
- •Normal blood pressure (BP) and heart rate (HR), or near normal BP and HR not considered clinically significant (NCS) by the Investigator at the Screening Day -14 (Visit 1) and Day -7 (Visit 2)
- •Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to the performance of any study-related procedures
- •Willingness and ability to comply with schedule for follow-up visits and postoperative evaluations
排除标准
- •CNV or macular edema in the Study Eye secondary to any causes other than AMD
- •Any condition preventing visual acuity improvement in the Study Eye, e.g., fibrosis, atrophy, or retinal pigment epithelial tear involving the center of the macula
- •Any ophthalmic condition that precludes adequate ophthalmic examination or requires treatment
- •Retinal detachment or history of retinal detachment in the Study Eye
- •Active uncontrolled glaucoma with intraocular pressure (IOP) ≥ 30 mmHg despite treatment with more than 2 glaucoma medications, advanced glaucoma with cup-to-disc ratio of ≥ 0.9, visual field defects secondary to glaucoma that involve the macula, and/or optic atrophy from glaucoma
- •History of intravitreal therapy in the Study Eye, such as intravitreal steroid injection or an investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening
- •History of vitrectomy, trabeculectomy, glaucoma tube-shunt procedure, minimally invasive glaucoma surgery (MIGS) device, or other filtration surgery in the Study Eye
- •Photodynamic therapy or retinal laser for the treatment of wAMD within the last 6 months
- •Any prior therapeutic radiation in the region of the Study Eye such as whole brain radiation, proton beam radiation, gamma knife treatment, or plaque brachytherapy
- •Any previous intraocular or refractive surgery on the Study Eye within 6 months
- •Any previous gene therapy in the Study Eye
- •Presence of an ocular implant in the Study Eye at Screening, excluding intraocular lens and custom flex iris prosthesis
- •Any diabetic retinopathy or other retinal vascular disease including retinal vein occlusion, retinal artery occlusion, retinal arterial macro aneurysm, ocular ischemic syndrome, retinal vasculitis, vitritis, posterior uveitis
- •Medically uncontrolled diabetes, defined as HbA1C > 8.0
- •History of ocular melanoma
- •History of any known inherited retinal disease
- •Currently taking any anticoagulant therapy, which is deemed medically necessary and cannot be permanently stopped at least 2 weeks prior to NG101 injection, excluding prophylactic low-dose aspirin therapy
- •Any underlying systemic diseases as unstable or severe cardiovascular, cerebrovascular, dementia or neurodegenerative diseases of a level that prevents adequate evaluation of the subject during the study, active malignancy or currently undergoing treatment for active malignancy at Screening or a history of malignancy that precludes completion of this 260 week study, and immunocompromised conditions and/or need for immunosuppressive therapy
- •Active hepatitis B or C
- •History of human immunodeficiency virus (HIV), active tuberculosis, and/or syphilis
- •Any significant illness that would preclude study compliance and follow-up
- •Subjects who, in the Investigator's opinion, lack the mental capacity to provide written informed consent for study participation
研究组 & 干预措施
NG101 Gene Therapy Group 2
Experimental
Single subretinal injection of 3x10^9 vector genomes of NG101 AAV gene therapy
干预措施: NG101 AAV gene therapy (Genetic)
NG101 Gene Therapy Group 3
Experimental
Single subretinal injection of 8x10^9 vector genomes of NG101 AAV gene therapy
干预措施: NG101 AAV gene therapy (Genetic)
NG101 Gene Therapy Group 1
Experimental
Single subretinal injection of 1x10^9 vector genomes of NG101 AAV gene therapy
干预措施: NG101 AAV gene therapy (Genetic)
结局指标
主要结局
Adverse Events
时间窗: From Day 0 (pre-treatment) through Week 24
Incidence and severity of ocular and non-ocular adverse events (AEs) for each cohort
次要结局
- Ocular and Non -Ocular Adverse Events after week 24(From Day 0 (pre-treatment) and through Weeks 52, 104, 156, 208 and 260)
- Systemic Immunogenic Response(From Day 0 (pre-treatment) to Weeks 24, 52, and 104)
- Signs of CNV (choroidal neovascularization) Activity(From Day -7 to Weeks 8, 24, 52, and 104)
- Central Subfield Thickness (CST)(From Day -7 to Weeks 24, 52, 104, 156, 208, and 260)
- Best Corrected Visual Acuity (BCVA)(From Day -7 to Weeks 24, 52, 104, 156, 208 and 260)
- Cumulative Number of Rescue Therapy Injections(Through Weeks 24, 52, 104, 156, 208, and 260)
研究者
研究点 (14)
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