中国临床试验注册中心 - 世界卫生组织国际临床试验注册平台一级注册机构

注册号： Registration number：	ChiCTR2400094879
最近更新日期： Date of Last Refreshed on：	2026-01-19 15:05:25
注册时间： Date of Registration：	2024-12-30 00:00:00
注册号状态：	预注册
Registration Status：	Prospective registration
注册题目：	一项在湿性（新生血管）年龄相关性黄斑变性（wAMD）患者中评价玻璃体腔内注射EXG202注射液的安全性和初步有效性的开放性、剂量递增及扩展的临床研究
Public title：	An open-label, dose-escalation, and dose-expansion study to evaluate the safety and preliminary efficacy of intravitreal EXG202 injection in patients with wet (neovascular) age-related macular degeneration (wAMD)
注册题目简写：
English Acronym：
研究课题的正式科学名称：	一项在湿性（新生血管）年龄相关性黄斑变性（wAMD）患者中评价玻璃体腔内注射EXG202注射液的安全性和初步有效性的开放性、剂量递增及扩展的临床研究
Scientific title：	An open-label, dose-escalation, and dose-expansion study to evaluate the safety and preliminary efficacy of intravitreal EXG202 injection in patients with wet (neovascular) age-related macular degeneration (wAMD)
研究课题代号(代码)： Study subject ID：
在二级注册机构或其它机构的注册号： The registration number of the Partner Registry or other register：

申请注册联系人：	王莉菲	研究负责人：	王莉菲
Applicant：	Wang Lifei	Study leader：	Wang Lifei
申请注册联系人电话： Applicant telephone：	+86 319 323 7196	研究负责人电话： Study leader's telephone：	+86 319 3237196
申请注册联系人传真： Applicant Fax：		研究负责人传真： Study leader's fax：
申请注册联系人电子邮件： Applicant E-mail：	wlfhb@126.com	研究负责人电子邮件： Study leader's E-mail：	wlfhb@126.com
申请单位网址(自愿提供)： Applicant website(voluntary supply)：		研究负责人网址(自愿提供)： Study leader's website(voluntary supply)：
申请注册联系人通讯地址：	河北省邢台市泉北东大街399号	研究负责人通讯地址：	河北省邢台市泉北东大街399号
Applicant address：	No. 399, Quanbei East Avenue, Xingtai City, Hebei Province	Study leader's address：	No. 399, Quanbei East Avenue, Xingtai City, Hebei Province
申请注册联系人邮政编码： Applicant postcode：		研究负责人邮政编码： Study leader's postcode：
申请人所在单位：	河北省眼科医院
Applicant's institution：	Hebei Eye Hospital
研究负责人所在单位：	河北省眼科医院
Affiliation of the Leader：	Hebei Eye Hospital

是否获伦理委员会批准：	是
Approved by ethic committee：	Yes
伦理委员会批件文号： Approved No. of ethic committee：	YK-KY202400202	伦理委员会批件附件： Approved file of Ethical Committee：	查看附件View
批准本研究的伦理委员会名称：	河北省眼科医院临床试验伦理委员会
Name of the ethic committee：	Hebei Eye Hospital clinical Trial Ethics Committee
伦理委员会批准日期： Date of approved by ethic committee：	2024-11-25 00:00:00
伦理委员会联系人：	张晓
Contact Name of the ethic committee：	Zhang Xiao
伦理委员会联系地址：	河北省邢台市泉北东大街399号
Contact Address of the ethic committee：	No. 399, Quanbei East Avenue, Xingtai City, Hebei Province
伦理委员会联系人电话： Contact phone of the ethic committee：	+86 319 3237720	伦理委员会联系人邮箱： Contact email of the ethic committee：	hbsykyyll@126.com

研究实施负责（组长）单位：

河北省眼科医院

Primary sponsor：

Hebei Eye Hospital

研究实施负责（组长）单位地址：

河北省邢台市泉北东大街399号

Primary sponsor's address：

No. 399, Quanbei East Avenue, Xingtai City, Hebei Province

试验主办单位(项目批准或申办者)：

Secondary sponsor：

国家：	中国	省(直辖市)：	河北	市(区县)：
Country：	China	Province：	Hebei	City：
单位(医院)：	河北省眼科医院	具体地址：	河北省邢台市泉北东大街399号
Institution hospital：	Hebei Eye Hospital	Address：	No. 399, Quanbei East Avenue, Xingtai City, Hebei Province

经费或物资来源：

广州嘉因生物科技有限公司

Source(s) of funding：

Guangzhou Jiayin Biotech Ltd

研究疾病：

年龄相关性黄斑变性

Target disease：

Age-related macular degeneration

研究疾病代码：

Target disease code：

研究类型：

干预性研究

Study type：

Interventional study

研究所处阶段：

其它

Study phase：

N/A

研究设计：

单臂

Study design：

Single arm

研究目的：

主要目的：安全性和耐受性主要安全性终点: 0~24周眼部和非眼部的不良事件(AE)、严重不良事件(SAE)的类型、严重程度及发生率，探索最大耐受剂量(MTD)。次要安全性终点：0~52周眼部和非眼部的不良事件(AE)、严重不良事件(SAE)的类型、严重程度及发生率;免疫安全性;病毒脱落;ABD-VEGFR融合蛋白在血清中的浓度变化。次要目的：有效性主要有效性终点：52周BCVA较基线变化；次要有效性终点：其他各访视BCVA较基线变化:各访视CRT较基线变化；补救治疗次数。探索性目的：药效学特征，ABD-VEGFR融合蛋白在房水中的浓度变化。

Objectives of Study：

Primary Objective: Safety and tolerability Main safety endpoints: The type, severity and incidence of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs) at 0~24 weeks, and the maximum tolerated dose (MTD) was explored. Secondary safety endpoints: type, severity and incidence of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs) at 0~52 weeks; immune safety; virus shedding; Changes in the concentration of ABD-VEGFR fusion protein in serum. Secondary purpose: Effectiveness Primary Effectiveness Endpoint: Change from baseline in BCVA at 52 weeks; Secondary Effectiveness Endpoints: Change from Baseline in BCVA at each other visit: Change from baseline in CRT at each visit; Number of salvage treatments. Exploratory objective: Pharmacodynamic characterization, change in concentration of ABD-VEGFR fusion protein in aqueous humor.

药物成份或治疗方案详述：

Description for medicine or protocol of treatment in detail：

纳入标准：

Inclusion criteria

1. Male or female, age >=50 years old;
2. The study eye must meet the following criteria: 
A. Confirmed diagnosis of wAMD with active lesions, defined as the presence of any of the following lesions in the macular area by OCT:
a) Intraretinal hemorrhage or fluid; 
b) subretinal fluid;
c) subretinal hemorrhage; 
B. BCVA scores detected at screening using the ETDRS visual acuity chart (see Appendix 3 for details) must be between 73-19 letters (including cut-off values) (equivalent to 20/40 to 20/400 of Snellen's visual acuity);
3. Clinically significant response to standard anti-VEGF therapy within 6 months prior to screening or during the screening lead-in period. A clinically significant response is defined as a decrease in retinal thickness, subretinal effusion, or intraretinal fluid that meets at least one of the following:  
a) at least 50 μm improvement if CRT is less than 400 μm; 
b) at least 15% improvement if CRT is above 400 microns; 
c) Subretinal effusion or intraretinal effusion as assessed by the investigator is less than before treatment.
4. Subjects have clear refractive media and adequate pupil dilation at screening in order to obtain high-quality retinal images to confirm the diagnosis;
5. Subjects (including male subjects) have no pregnancy plan during the screening period and the whole trial period and voluntarily take effective contraceptive measures and have no sperm donation and egg donation plans;
6. Voluntarily participate in this clinical trial, understand the study procedures and sign the informed consent form before screening; Good compliance with study procedures.

排除标准：

1.研究眼存在除wAMD以外的可能影响中心视力和/或黄斑检测的任何眼部疾病（如黄斑裂孔、黄斑前膜等）； 2.研究眼既往有视网膜脱离病史或筛选期存在视网膜脱离（如孔源性视网膜脱离、牵拉性视网膜脱离等）； 3.研究眼存在wAMD以外的原因引起的MNV（如糖尿病视网膜病变、病理性近视、视网膜静脉阻塞、血管样条纹病变、眼组织胞浆菌病、外伤等）、与wAMD无关的黄斑病理学病史； 4.研究眼计划在研究期间接受任何眼内手术； 5.研究眼视网膜下出血累计中心凹，且出血面积>=4个DA（视盘面积）； 6.研究眼现患>=1+玻璃体出血； 7.任意眼存在与眼科手术无关的特发性或自身免疫相关性葡萄膜炎病史； 8.任意眼活动性眼部感染（如结膜炎、角膜炎、巩膜炎、眼内炎、睑缘炎、视网膜视神经炎等）； 9.研究眼现患视网膜血管瘤增生（RAP）、中心性浆液性脉络膜视网膜病变或有症状的玻璃体黄斑牵引综合症； 10.存在青光眼或视神经病变，涉及或危及研究眼的中心视野或研究眼存在未控制的高眼压，定义为目前正在接受>=2种降眼压药物； 11.既往发生过继发于激素治疗的眼压升高（定义为：接受局部、眼周、IVT或系统性激素用药后30天内，发生眼压＞22 mmHg或者需要使用降眼压药物对症处理）； 12.研究眼屈光不正的球镜当量显示近视>8屈光度； 13.存在以下疾病： a)不稳定或严重的心血管疾病，脑血管意外或短暂性脑缺血发作（筛选前6个月内）、心肌梗死（筛选前6个月内）、不稳定型心绞痛、充血性心力衰竭或需药物治疗的严重心律失常或严重肢体缺血； b)未受控制的高血压，定义为经降压药物充分治疗后收缩压>160 mmHg，舒张压>100 mmHg； c)糖化血红蛋白（HbA1c）>= 7.0 %； d)筛选前 12 个月内患有脑血管疾病，并经研究者判断不适合参与本试验或会影响对受试者的评估； e)存在神经退行性疾病（例如，阿尔茨海默病、帕金森病），并经研究者判断会影响研究者对受试者的评估； f)首次给药前5年内有恶性肿瘤史者（以下肿瘤疾病除外：皮肤基底细胞癌、皮肤鳞状上皮细胞癌、宫颈原位癌、乳腺原位癌、甲状腺原位癌、结直肠原位癌等经治疗已经得到控制者）； g)研究期间需要免疫抑制剂治疗的疾病； 14.肝功能异常者： a)丙氨酸氨基转移酶（ALT）>=3 × ULN（正常值上限）； b)天门冬氨酸氨基转氨酶（AST）>=3× ULN； 15.血清病毒学检查：乙型肝炎患者（【乙肝病毒表面抗原（HBsAg）阳性，且HBV-DNA 病毒载量>=2000 IU/mL】；丙型肝炎患者【若丙型肝炎病毒（HCV）抗体阳性，且丙型肝炎病毒-核糖核酸（HCV-RNA）>=1000 IU/ml】；梅毒筛查（Tp-Ab）阳性，且梅毒螺旋体DNA阳性；已知人类免疫缺陷病毒（HIV）阳性病史或HIV筛查阳性； 16.目前或既往使用过任何已知对视网膜或视神经有毒性的药物，包括但不限于氯喹/羟氯喹、去氧胺、吩噻嗪和乙胺丁醇； 17.研究眼有青光眼分流术、小梁切除术或小切口青光眼手术史； 18.研究眼有角膜移植或其他角膜手术史; 19.研究眼在筛选前30天内有过YAG, SLT或ALT激光治疗。 20.研究眼既往接受过PDT或热激光治疗nAMD； 21.研究眼既往6个月内接受过地塞米松玻璃体内植入剂（傲迪适，Ozurdex）或3年内接受过氟轻松玻璃体内植入剂（优施莹，Iluvien）治疗；或筛选前6个月内接受过玻璃体内注射激素或其他非抗-VEGF产品； 22.非研究眼3个月内接受过地塞米松玻璃体内植入剂（Ozurdex）或3年内接受过氟轻松玻璃体内植入剂（优施莹，Iluvien）治疗； 23.对荧光素钠、吲哚菁绿有过敏反应或过敏史； 24.既往接受过任何眼部或全身基因治疗； 25.筛选前90天内（或试验药物5个半衰期内，以时间长者为准）参加过任何其他临床试验，且已接受试验药物的受试者（研究药物为维生素、矿物质除外）； 26.妊娠期或哺乳期女性； 27.研究者认为不适合参与本试验的受试者（如无法理解和遵从试验要求或安全性考虑认为不适合者）。

Exclusion criteria：

1. Presence of any ocular disease (such as macular hole, epimacular membrane, etc.) in the study eye other than wAMD that may affect central vision and/or macular detection; 2. The study eye has a history of retinal detachment in the past or has retinal detachment during the screening period (such as rhegmatogenous retinal detachment, traction retinal detachment, etc.); 3. History of macular pathology unrelated to wAMD due to MNV caused by reasons other than wAMD in the study eye (such as diabetic retinopathy, pathological myopia, retinal vein occlusion, vascular streak lesion, ocular histoplasmosis, trauma, etc.); 4. The study eye is planned to undergo any intraocular surgery during the study; 5. The subretinal hemorrhage of the study eye accumulated in the fovea, and the hemorrhage area was >=4 DA (optic disc area); 6. Current > in the study eye = 1 + vitreous hemorrhage; 7. History of idiopathic or autoimmune-associated uveitis unrelated to ophthalmic surgery in any eye; 8. Active ocular infection in any eye (such as conjunctivitis, keratitis, scleritis, endophthalmitis, blepharitis, retinal optic neuritis, etc.); 9. Study eye with retinal angiomatosis hyperplasia (RAP), central serous chorioretinopathy or symptomatic vitreomacular traction syndrome; 10. Presence of glaucoma or optic neuropathy involving or jeopardizing the central visual field of the study eye or presence of uncontrolled high intraocular pressure in the study eye, defined as currently receiving >=2 intraocular hypotensive drugs; 11. Previous intraocular hypertension secondary to hormonal therapy (defined as: intraocular pressure > 22 mmHg within 30 days after receiving topical, periocular or IVT or systemic hormone therapy or the need for symptomatic treatment with intraocular hypotensive drugs); 12. The spherical equivalent of the refractive error of the study eye showed a myopic >8 diopters; 13. Presence of the following diseases: a) Unstable or severe cardiovascular disease, cerebrovascular accident or transient ischemic attack (within 6 months prior to screening), myocardial infarction (within 6 months prior to screening), unstable angina, congestive heart failure or severe arrhythmia or severe limb ischemia requiring medication; b) Uncontrolled hypertension, defined as systolic blood pressure > 160 mmHg and diastolic blood pressure > 100 mmHg after adequate treatment with antihypertensive drugs; c) glycosylated hemoglobin (HbA1c) > = 7.0 %; d) Cerebrovascular disease within 12 months prior to screening, which is judged by the investigator to be unsuitable for participation in this trial or will affect the evaluation of the subject; e) the presence of neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease) that, in the judgment of the investigator, would affect the investigator's evaluation of the subject; f) Those who have a history of malignant tumors within 5 years before the first dose (except for the following tumor diseases: basal cell carcinoma of the skin, squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, carcinoma in situ of the thyroid, carcinoma in situ of the colorectum, etc., which have been controlled by treatment); g) Disease requiring immunosuppressant therapy during the study period; 14. Patients with abnormal liver function: a) alanine aminotransferase (ALT) >=3 × ULN (upper limit of normal); b) aspartate aminotransferase (AST) >=3× ULN; 15. Serovirological examination: Patients with hepatitis B ([hepatitis B virus surface antigen (HBsAg)-positive and HBV-DNA viral load >=2000 IU/mL]; Patients with hepatitis C (if hepatitis C virus (HCV) antibody is positive and hepatitis C virus-ribonucleic acid (HCV-RNA) >=1000 IU/ml); Positive syphilis screening (Tp-Ab) with Treponema pallidum DNA; Known history of human immunodeficiency virus (HIV) positivity or positive HIV screening; 16. Current or prior use of any drug known to be toxic to the retina or optic nerve, including but not limited to chloroquine/hydroxychloroquine, deoxyamine, phenothiazine, and ethambutol; 17. History of glaucoma shunt, trabeculectomy, or small-incision glaucoma surgery in the study eye; 18. History of corneal transplantation or other corneal surgery in the study eye; 19. The study eye has had YAG, SLT, or ALT laser treatment within 30 days prior to screening. 20. Previous treatment of nAMD with PDT or thermal laser in the study eye; 21. The study eye has been treated with dexamethasone intravitreal implant (Ozurdex) within 6 months or fluocinolone acetonide intravitreal implant (Iluvien) within 3 years; or received intravitreal injection of corticosteroids or other non-anti-VEGF products within 6 months prior to screening; 22. Treatment with dexamethasone intravitreal implant (Ozurdex) within 3 months or fluocinolone acetonide intravitreal implant (Iluvien) within 3 years in the non-study eye; 23. Allergic reaction or history of allergy to sodium fluorescein and indocyanine green; 24. Prior treatment with any ocular or systemic gene therapy; 25. Subjects who have participated in any other clinical trial within 90 days before screening (or within 5 half-lives of the trial drug, whichever is longer) and have received the trial drug (except for vitamins and minerals); 26. Pregnant or lactating females; 27. Subjects who are considered unsuitable to participate in this trial by the investigator (such as those who are unable to understand and comply with the requirements of the trial or who are considered unsuitable for safety considerations).

研究实施时间：

Study execute time：

从 From 2025-01-01 00:00:00至 To 2026-09-30 00:00:00

征募观察对象时间：

Recruiting time：

从 From 2025-01-01 00:00:00 至 To 2025-09-30 00:00:00

干预措施：

Interventions：

组别：	EXG202注射液试验组	样本量：	24
Group：	EXG202 injection Group	Sample size：	24
干预措施：	EXG202注射液玻单次玻璃体腔注射	干预措施代码：
Intervention：	EXG202 injection single intravitreal injection	Intervention code：

研究实施地点：

Countries of recruitment and research settings：

国家：	中国	省(直辖市)：	河北	市(区县)：
Country：	China	Province：	Hebei	City：
单位(医院)：	河北省眼科医院	单位级别：	三甲
Institution hospital：	Hebei Eye Hospital	Level of the institution：	Tertiary A

测量指标：

Outcomes：

指标中文名：	最佳矫正视力（BCVA）	指标类型：	次要指标
Outcome：	Best Corrected Visual Acuity, BCVA	Type：	Secondary indicator
测量时间点：	治疗后52周（W52）	测量方法：	采用早期治疗糖尿病视网膜病变研究（ETDRS）视力表进行评估
Measure time point of outcome：	52 weeks after treatment	Measure method：	Evaluation using the Early Treatment of diabetes Retinopathy Study (ETDRS) visual acuity chart

指标中文名：	评估治疗后至52周眼部和非眼部的不良事件（AE）、严重不良事件（SAE）的类型	指标类型：	主要指标
Outcome：	Assess treatment-emergent adverse events (AEs) and serious adverse events (SAEs) through Week 52 by organ system and incidence	Type：	Primary indicator
测量时间点：	治疗后52 周	测量方法：	根据原始记录进行评估
Measure time point of outcome：	52 weeks after treatment	Measure method：	Evaluate based on original records

采集人体标本：

Collecting sample(s)
from participants：

标本中文名：	血清	组织：
Sample Name：	Serum	Tissue：
人体标本去向	使用后销毁	说明
Fate of sample：	Destruction after use	Note：

标本中文名：	血液	组织：
Sample Name：	Blood	Tissue：
人体标本去向	使用后销毁	说明
Fate of sample：	Destruction after use	Note：

征募研究对象情况：

Recruiting status：

尚未开始

Not yet recruiting

年龄范围：

Participant age：

最小 Min age	50	岁 years
最大 Max age		岁 years

性别：

男女均可

Gender：

Both

随机方法（请说明由何人用什么方法产生随机序列）：

无

Randomization Procedure (please state who generates the random number sequence and by what method)：

None

是否公开试验完成后的统计结果:

Calculated Results after the Study Completed public access:

不公开/Private

盲法：	无
Blinding：	None

是否共享原始数据： IPD sharing	否No
共享原始数据的方式（说明：请填入公开原始数据日期和方式，如采用网络平台，需填该网络平台名称和网址）：	不共享
The way of sharing IPD”(include metadata and protocol, If use web-based public database, please provide the url)：	none
数据采集和管理（说明：数据采集和管理由两部分组成，一为病例记录表(Case Record Form, CRF)，二为电子采集和管理系统(Electronic Data Capture, EDC)，如ResMan即为一种基于互联网的EDC：	电子病例报告表
Data collection and Management (A standard data collection and management system include a CRF and an electronic data capture：	eCRF
数据与安全监察委员会： Data and Safety Monitoring Committee：	无/No
注册人： Name of Registration：	2024-12-30 11:39:08