Clinical Research of Human Retinal Pigment Epithelial (HuRPE) Cell Injection on Atrophy of High Myopia Macular Area
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 9
- 主要终点
- Number of subjects with Local and systemic adverse events
研究概览
简要总结
To evaluate the safety and tolerability of human retinal pigment epithelial (HuRPE) cell injection subretinal transplantation for atrophy of high myopia macular area, and to explore the maximum tolerated dose (MTD).
详细描述
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of HuRPE cells in subjects with Macular degeneration caused by high myopia. The investigators will recruit and enroll 9 patients based on specific inclusive/exclusive criteria. Experimental and self-controlled eye will be determined based on best-corrected visual acuity (BCVA). The eye with BCVA between 5 and 60 ETDRS letters will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of HuRPE cells (300.000, 500,000 or 1,000,000) respectively, while the other one as control eye, will not receive the surgery.
HuRPE cells will be obtained from Eyecure Therapeutics, Inc.(Jiangsu) located in WuXi, Jiangsu Province. The obtained HuRPE cells will meet its quality standards and conform to Good manufacturing practices (GMP). HuRPE cells will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects after transplantation. Dosage and time duration of immunosuppressive agents will be regulated strictly relying on the condition of immune rejection. Subjects will be monitored with ophthalmologic and systemic examinations frequently at regular post-transplant intervals after HuRPE cells transplantation.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A patient who is able (or with the assistance of his or her legal representative) to read and understand the informed consent form and is willing to sign a written informed consent;
- •age ≥ 18 years old, male or female;
- •myopic diopter ≥ -6D or visual axis length ≥ 26mm;
- •study the best corrected visual acuity ≥ 5 letters, ≤ 60 letters (using ETDRS eye chart);
- •According to the researcher's judgment, the patient's high myopia macular atrophy is the main reason for the patient's vision loss;
- •If both eyes meet the inclusion criteria, the selected disease is more severe; the eyes are the same, and the right eye is the study eye.
排除标准
- •The study eye has intravitreal hemorrhage within 2 months before the group visit;
- •the study of the eye involved in the foveal scar, fibrosis, suggesting a serious irreversible vision loss;
- •The study eye diagnosis is a pupillary afferent defect (APD);
- •the study eye has significant refractive interstitial opacity, and moderate to severe cataract, may cause interference to OCT and fundus examination;
- •any eye diagnosed as infectious blepharitis, keratitis, scleritis or conjunctivitis; or there are currently serious systemic infectious diseases;
- •Uncontrollable glaucoma, defined as a patient who is adequately treated with medication, with an intraocular pressure still above 25 mmHg, or who has undergone previous or filtering surgery;
- •Study the eye with other diseases that affect vision;
- •traumatic macular hole, idiopathic macular hole, combined with other parts of the retinal tear;
- •single eye blind patients;
- •The study eye is an aphakic eye (except for the intraocular lens);
- •Any eye has a history of idiopathic or autoimmune-related uveitis;
- •Any eye is diagnosed as an iris neovascularization;
- •Received cataract surgery within 2 months prior to enrollment;
- •Other intraocular surgery was performed within 3 months prior to the group visit;
- •Have an allergic reaction or allergic history to sodium fluorescein, a history of allergies to protein products for therapeutic or diagnostic use, and allergies to two or more drugs and/or non-drug factors, or those with allergic diseases;
- •Diabetes patients with uncontrolled blood glucose (fasting blood glucose ≥7.0mmol/L or 2h postprandial blood glucose ≥11.1mmol/L);
- •There is a history of surgery within 1 month before screening, or there are currently unhealed wounds, ulcers, fractures, etc.;
- •patients with uncontrolled hypertension, defined as the best treatment plan, a single measurement of systolic blood pressure > 180mmHg, two consecutive measurements of systolic blood pressure > 160mmHg or diastolic blood pressure > 100mmHg;
- •History of myocardial infarction within 6 months prior to enrollment;
- •There is active diffuse intravascular coagulation or obvious bleeding tendency before screening. Anticoagulation or anti-platelet aggregation drugs are used in addition to aspirin/non-steroidal anti-inflammatory drugs within 14 days before screening;
- •Any clinical problem that cannot be controlled (such as severe mental, neurological, cardiovascular, respiratory, malignant, Parkinson's, Alzheimer's disease, etc.);
- •Women of childbearing age were positive in the screening visit pregnancy test. Women who wish to breastfeed during the study. All women of childbearing age (male and female) did not agree to take effective contraceptive measures (such as intrauterine devices, birth control pills or condoms) during the entire study period and within 30 days of the end of the visit period;
- •Patients who participated or are currently participating in other clinical studies within 30 days prior to screening;
- •Hemoglobin <100 g/L, blood Plt≤100×109/L, neutrophil count <1.0×109/L, ALT/AST> upper limit of normal value (ULN)×1.5, Cr>1.3 mg/dL;
- •Researchers believe that patients who are not eligible for inclusion, or other medical conditions, limit subject compliance, safety, or impact study results.
结局指标
主要结局
Number of subjects with Local and systemic adverse events
时间窗: six months following transplantation
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
次要结局
- Mean change of Best-Corrected Visual Acuity (BCVA)(six months following transplantation)
- Change in NEI VFQ-25 Total Score(six months following transplantation)
- Evidence of successful engraftment(six months following transplantation)
研究者
Xiaodong Sun
Professor
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine