NCT07063030招募中1 期
An Exploratory Clinical Study to Evaluate LX107 Gene Therapy in Patients With AIPL1 Biallelic Mutation-related Inherited Retinal Dystrophy (AIPL1-IRD)
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine1 个研究点 分布在 1 个国家目标入组 13 人开始时间: 2025年7月15日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
研究概览
简要总结
Administering subretinal injection of LX107 injection (a gene therapy drug) to patients with retinal dystrophy caused by AIPL1 gene mutation to evaluate its efficacy and safety.
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
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The subject and/or their guardian signs a written informed consent form and is willing to comply with the long-term follow-up protocol and supporting protocols.
- •Adult or pediatric patients (aged ≥ 4 years) diagnosed with AIPL1-IRD.
- •Definitive molecular diagnosis of biallelic AIPL1 gene mutations confirmed by next-generation sequencing combined with Sanger validation.
- •The study eye has a best-corrected visual acuity of no more than 58 letters (approximately equivalent to decimal visual acuity ≤ 0.3) using the ETDRS visual acuity chart at baseline.
- •Note: Only one eye will be designated as the "study eye" (i.e., the eye to receive treatment) at the investigator's discretion.
排除标准
- •For any eye with the following conditions:
- •A history of ocular diseases that, in the investigator's judgment, may hinder the planned treatment or interfere with the interpretation of study endpoints (e.g., glaucoma, diabetic retinopathy, retinal vein occlusion, retinal detachment, posterior or panuveitis, etc.).
- •Any eye with a history of gene therapy for IRD or other hereditary neuro-ophthalmic diseases (including but not limited to other viral vector-based gene therapies, mRNA therapies, etc.).
- •A lack of sufficient viable retinal cells as determined by non-invasive methods such as OCT or ophthalmoscopy.
- •Any active intraocular or periocular infection in the study eye (e.g., infectious conjunctivitis, keratitis, scleritis, endophthalmitis, infectious blepharitis, uveitis).
- •A history of intraocular surgery (e.g., vitrectomy, cataract surgery, trabeculectomy, or other filtering surgery) in the 6 months prior to the screening visit.
- •For any systemic conditions:
- •Uncontrolled hypertension, defined as systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg. If the initial measurement exceeds the above limits, it may be repeated on the same day or another day during the screening period; if the subject is taking oral antihypertensive drugs, they must have been on a stable dose of the same drug for at least 30 days prior to screening.
- •Diabetic patients meeting any of the following criteria: ① known to have macrovascular complications; ② baseline HbA1c > 7.5%; ③ receiving treatment with two or more oral hypoglycemic agents, insulin, or GLP-1 receptor agonists.
- •A history of any other diseases, metabolic disorders, physical examination findings, or clinical laboratory abnormalities that, in the investigator's judgment, may contraindicate the use of the study drug, interfere with the interpretation of study results, or place the subject at high risk of treatment complications, including but not limited to: AIDS, syphilis, acute/chronic active hepatitis B or C, coagulation disorders, a history of treated or untreated malignancy within the past 5 years (except for localized basal cell carcinoma of the skin or in situ cervical cancer), etc.
- •Any of the following laboratory abnormalities: ① platelet count < 100 × 10⁹/L, hemoglobin (Hb) < 10 g/dL (males) or < 9 g/dL (females); ② aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 × ULN; ③ serum creatinine or urea > 1.5 × ULN.
- •Administration of any other investigational drug (except vitamins and minerals) within 3 months prior to screening, or an attempt to participate in another clinical trial during the study period.
- •Other circumstances deemed unsuitable for enrollment by the investigator.
- •Unwillingness to use effective contraceptive methods during the study; pregnant or lactating women, or women planning to become pregnant or lactate during the study period.
研究组 & 干预措施
Intervention
Experimental
Experimental: LX107 Injection
Arm Description:
- Low dose group: subretinal injection of LX107 -3×10⁹ VG/eye
- Medium dose group: subretinal injection of LX107- 1×10¹⁰ VG/eye
- High dose group: subretinal injection of LX107 -3×10¹⁰ VG/eye
干预措施: LX107 Injection (Genetic)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: 6 Months
Incidence of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs) following LX107 subretinal injection
次要结局
- Changes in Functional Magnetic Resonance Imaging (fMRI) Results(6 months、12 months)
- Mean Change from Baseline in Multi-luminance Mobility Test (MLMT)(6 months、12 months)
- Mean Change from Baseline in Full-Field Sensitivity Threshold (FST)(6 months、12 months)
- Mean Change from Baseline in Visual Acuity (VA)(6 months、12 months)
- Mean Change from Baseline in Visual Fields(6 months、12 months)
研究者
Xiaodong Sun
Director
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
研究点 (1)
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