NCT06345898招募中早期 1 期
Clinical Study on the Safety and Efficacy of JWK002 Injection Administered as a Single Subretinal Injection in Subjects With X-linked Retinoschisis
适应症
干预措施
相关药物
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Safety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)
研究概览
简要总结
This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 5 Years 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Fully understand the purpose and requirements of the trial, voluntarily participate in the clinical trial and sign the informed consent, and be able to cooperate with the testing required in various studies according to the requirements of the program;
- •Male, ≥5 years old and < 18 years old (subject to the date of signing the informed consent);
- •Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;
- •Best corrected visual acuity (BCVA) of the study eye ≤63 letters (equivalent to Snellen 20/63); Best corrected visual acuity (BCVA) ≥34 letters (equivalent to Snellen 20/200).
排除标准
- •Carbonic anhydrase inhibitor treatment within 3 months;
- •The presence of other conditions in the study eye that may cause vision loss (e.g., atrophy of the optic nerve, advanced glaucoma, uveitis, the presence of vesicular splitting chambers or vesicular retinal detachment that may affect the subretinal injection operator);
- •Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity;
- •There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint;
- •Patients who had undergone internal eye surgery within 6 months;
- •Patients with AAV8 neutralizing antibody titer ≥1:1000;
- •Have previously received any gene therapy or cell therapy;
- •fertile subjects are unwilling to use contraception;
- •The presence of any of the following: an active infection that the investigator believes may affect the patient's participation in the study or affect the study results that requires systematic treatment; Hepatitis B surface antigen positive, hepatitis B virus deoxyribonucleic acid (HBV DNA) copy number > ULN; Hepatitis C virus (HCV) antibody positive and HCV-RNA copy number >ULN; Treponema pallidum antibody positive; Positive for human immunodeficiency virus (HIV) antibodies;
- •Malignant tumors (basal cell or squamous cell skin cancer) were diagnosed within 5 years prior to screening;
- •has or has had a systemic immune system disease;
- •Laboratory values considered to be clinically significant were abnormal: alanine and/or aspartate transaminase >2.5×ULN, total bilirubin >1.5×ULN, serum creatinine >1.5×ULN, prothrombin time ≥1.5×ULN, activated partial thromboplastin time ≥1.5×ULN; (13) have a severe allergy or a known allergy to the drugs used in the treatment or examination in the study protocol, including the investigational drug allergy;
- •(14) Other situations that the investigator considers inappropriate for participation in this study.
研究组 & 干预措施
JWK002 at Low Dose Group
Experimental
Subretinal injection low dose of JWK002 in one eye
干预措施: JWK002 (Drug)
JWK002 at Medium Dose Group
Experimental
Subretinal injection medium dose of JWK002 in one eye
干预措施: JWK002 (Drug)
结局指标
主要结局
Safety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)
时间窗: baseline to day 7, day 14, month 1, 3, 6, 12
The primary outcome measures are safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
次要结局
- Best corrected visual acuity (BCVA)(baseline to day 7, day 14, month 1, 2, 3, 6, 12)
- Full-field electroretinogram(ff-ERG)(baseline to month 1, 3, 6, 12)
- Macular structure as assessed by swept source optical coherence tomography(baseline to month 1, 3, 6, 12)
- Visual field(baseline to month 1, 3, 6, 12)
研究者
研究点 (1)
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