Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of ZM-02 in Retinitis Pigmentosa
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Incidence of adverse events and serious adverse events
研究概览
简要总结
This is zM-02's safety, tOlerability, and efficacy in retinitis pigmentOsa first-in-humaN study (MOON). This trial is meant to evaluate the safety and efficacy of ZM-02 in Retinitis pigmentosa (RP) patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
详细描述
Retinitis pigmentosa (RP) is the most common inherited retinal disease. Individuals affected by RP often experience progressive visual impairment, potentially leading to legal blindness. There is currently no established effective clinical treatment available. We developed an innovative adeno-associated virus (AAV)-based gene therapy for individuals with RP, regardless of their causative mutations. Eight to twelve subjects with RP will be recruited and six to nine of them will receive a single unilateral intravitreal injection of ZM-02 at ascending doses, while two to three receive sham injections as the control group.
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
- Double (Participant, Care Provider)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who meet all of the following criteria can be selected as subjects:
- •Clinically diagnosed with retinal pigment degeneration
- •The visual acuity of study eye is no better than the finger counting, while the visual acuity of the study eye is not better than that of the contralateral eye
- •The subject has had visual experience above the finger counting
- •In the OCT examination of the tested eye, the disappearance of the ellipsoid zone is observed, but the inner nuclear layer and the nerve fiber layer of the retina are still present
- •The refractive power of the tested eye is between -6.00 D and +6.00 D
- •Not infected with the Human Immunodeficiency Virus (HIV) and other acute and chronic infectious diseases
- •Voluntarily sign an Informed Consent Form (ICF), and the age is not less than 18 years and not more than 65 years
- •Able to fully understand and agree to cooperate with the implementation of the research protocol
排除标准
- •Subjects who meet any one of the following exclusion criteria will be excluded from the study:
- •Pregnant women, breastfeeding women, or male and female subjects who do not agree to contraception during the 12 months before and after medication
- •Subjects with narrow anterior chamber angles or any other medical conditions that contraindicate pupil dilation
- •Subjects allergic to corticosteroids, who are unable to tolerate the corticosteroid treatment described in the protocol, or have active
- •concurrent infections that contraindicate treatment
- •Subjects with systemic diseases, or other medical or mental illnesses, or other safety concerns for the study
- •Subjects with other symptoms and/or diseases or conditions that can alter visual function, including but not limited to glaucoma and central nervous system lesions (mild cataracts are not included in this restriction)
- •Eye diseases that may interfere with the assessment of vision during the study and/or interfere with other ocular assessments such as OCT
- •Diseases that may affect the clinical trial, such as tumors, metabolic, immune-related diseases, etc.
- •Subjects who have undergone major eye surgery within the last 3 months before screening
- •Subjects with a history of malignant tumors within the last 5 years
- •Subjects with other retinal diseases not suitable for this study, such as retinal detachment
- •Patients undergoing or potentially undergoing immunosuppressive treatment for other diseases, excluding this study
- •Participation in any clinical trials other than this study within the last 3 months
- •Subjects who have received gene therapy outside of this study
- •Other reasons deemed by the researcher as unsuitable for participation in this study
研究组 & 干预措施
group 1
IVT administration of a single low dose ZM-02 injection
干预措施: ZM-02-L (Drug)
group 2
IVT administration of a single high dose ZM-02 injection
干预措施: ZM-02-H (Drug)
group 3
Sham IVT injection
干预措施: ZM-02-S (Procedure)
结局指标
主要结局
Incidence of adverse events and serious adverse events
时间窗: baseline to day 3, week 1, 4, 12, 24, 36, 52
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment. A serious adverse event (SAE) is any untoward medical occurrence at any dose that leading to the following: Results in death; Life-threatening, refers to an event in which the patient is at risk of death at the time of the event; it does not refer to an event which hypothetically might have caused death if it were more severe; Significant or permanent disability/incapacity, where disability refers to a serious disruption and damage of a person's ability to perform normal life functions; Requires inpatient hospitalization or prolongation of existing hospitalization; Congenital anomaly or birth defect; Other medically important events.
Changes in intraocular pressure (IOP) in Subjects
时间窗: baseline to day 3, week 1, 4, 12, 24, 36, 52
IOP refers to the fluid pressure inside the eye. Monitoring IOP ensures that interventions do not inadvertently increase IOP to dangerous levels. IOP will be measured using a clinical tonometry device.
次要结局
- Change of MLMT level(baseline to day 3, week 1, 4, 12, 24, 36, 52)
- Change of Quality of Life(baseline to day 3, week 1, 4, 12, 24, 36, 52)
- Change in best corrected visual acuity (BCVA)(baseline to day 3, week 1, 4, 12, 24, 36, 52)