NCT06706427进行中（未招募）1 期
A Phase I/II Study for Subretinal Injection of NGGT001 in Patients With Bietti Crystalline Corneoretinal Dystrophy
NGGT (Suzhou) Biotechnology Co., Ltd.4 个研究点 分布在 1 个国家目标入组 12 人开始时间: 2024年3月21日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
- 试验地点
- 4
- 主要终点
- Incidence of adverse events (AEs) from baseline to 52 weeks.
研究概览
简要总结
The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 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
- •Age ≥ 18 years old.
- •Male or female.
- •Confirmed diagnosis of BCD.
- •Molecular diagnosis confirmed cytochrome P450 family 4 subfamily v member 2 (CYP4V2) mutation.
- •AAV2 neutralizing antibody titer ≤1:
- •0.05 ≤ Best Corrected Visual Acuity (BCVA) ≤ 0.
- •6.00D ≤ Refractive error ≤ +3.00D.
- •Agree to take contraceptive measures from the start of the study until one year after medication administration.
- •Volunteer to participate in the study and sign informed consent.
排除标准
- •There are choroidal neovascularization or other ocular diseases caused by BCD, which are considered to affect the operation or interfere with the interpretation of clinical endpoint.
- •Patients with evidence of neovascularization or suspected neovascularization, and the presence of tubular reflectivity in the neuroepithelial layer as shown by OCT.
- •Those who had used any of the treatment drugs within 6 months before enrollment, such as Lucentis, Avastin, Conbercept, Triamcinolone acetonide, etc. These may affect the experimental observation.
- •The treated eyes have undergone intraocular surgery, such as photodynamic therapy (PDT), vitrectomy, periocular vascular bypass surgery, etc., or need intraocular surgery in the process of clinical research, such as cataract surgery, retinal laser therapy, etc.
- •Have used or may use systemic drugs that may cause eye damage, such as psoralen, tamoxifen, etc.
- •Highly sensitive or allergic to ingredients in the test drug (with allergic history of two or more drugs or food).
- •Physical examination, vital signs, and laboratory examination (such as blood routine, urine routine, blood biochemistry, coagulation function, immunology examination, etc.) are abnormal and clinically significant, or the investigators believe that the abnormal indicators have clinical significance.
- •There are diseases or medical histories that may affect drug safety or in vivo processes, especially cardiovascular, liver, kidney, endocrine, digestive tract, lung, nerve, blood, tumor, immune or metabolic disorders considered by investigators to be of clinical significance.
- •Participated in clinical trials of other drugs or medical devices within three months before enrollment.
- •Female patients who are pregnant or lactating.
- •Any other conditions which lead the investigator to determine the participant is unsuitable for this study.
研究组 & 干预措施
NGGT001
Experimental
Single Arm: This study is a single-arm design in which all participants receive the NGGT001 gene therapy administered via subretinal injection. Participants are divided into three dose-escalation groups to evaluate safety and efficacy.
干预措施: NGGT001 (Biological)
结局指标
主要结局
Incidence of adverse events (AEs) from baseline to 52 weeks.
时间窗: 52 weeks
To evaluate the incidence and severity of AEs, including serious AEs (SAEs) of subretinal injection of NGGT001 in patients with BCD.
Evaluate the improvement in BCVA compared to baseline at Week 12, 26 and 52.
时间窗: Week 12, Week 26 and Week 52
To evaluate the BCVA in ETDRS test of subretinal injection of NGGT001 from baseline to W12, 26 and 52.
次要结局
- Assessment of contrast sensitivity (CS) changes in dB compared to baseline at Week 12, 26 and 52.(Week 12, Week 26 and Week 52)
- Assessment of microperimetry changes in dB compared to baseline at Week 12, 26 and 52.(Week 12, Week 26 and Week 52)
- Assessment of Optical Coherence Tomography (OCT) retinal thickness changes compared to baseline at Week 12, 26 and 52.(Week 12, Week 26 and Week 52)
- Assessment of Multi-Luminance Mobility Test (MLMT) score changes compared to baseline at Week 12, 26 and 52.(Week 12, Week 26 and Week 52)
研究者
研究点 (4)
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