NCT04919473已完成1 期
A Phase I/IIa Open Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis Pigmentosa
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 11
- 试验地点
- 2
- 主要终点
- The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa
研究概览
简要总结
The purpose of the study is to evaluate the safety and tolerability of a single intravitreal injection of virally-carried Multi-Characteristic Opsin I (vMCO-I)
详细描述
This open label dose-escalation study evaluated 2 dose levels in up to 11 subjects of retinitis pigmentosa (3 in low dose and 8 in high dose per dose) with active vMCO-010. Subjects with confirmed diagnosis of Advanced Retinitis Pigmentosa (RP) based on clinical examination and dilated fundus examination, were considered for participation in this study. The primary endpoint for this study is safety and tolerability of vMCO-I at 16 weeks. All subjects were assessed for 52 weeks following treatment with vMCO-I
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
- 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
- •Diagnosis of advanced RP using Fundus Photographs
- •Clinical diagnosis of advanced retinal dystrophy
- •Prior documented (if any) retinal electrophysiological evidence of rod-cone photoreceptor degeneration
- •Snellen's visual acuity equivalent LP/NLP in worse (study) eye
- •Visual acuity in the non-study eye of no-better-than finger counting
- •Presence of retinal bipolar cells and retinal nerve fiber layer on OCT testing
排除标准
- •Prior participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy or any gene or stem cell therapy in the past six months.
- •Concurrent participation in another interventional clinical ocular study.
- •Pre-existing eye conditions such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, active uveitis, corneal or lenticular opacities).
- •Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function.
- •Subjects who are positive for hepatitis B, C, and HIV will be excluded.
- •Subjects who have undergone ocular surgery in the study eye within three months prior to Day
- •Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
- •Known sensitivity to any component of the study agent or medications planned for use in the peri-operative period.
- •Subjects will be excluded if immunological studies show presence of neutralizing antibodies to AAV2 above 1:
- •Presence of narrow iridocorneal angles contraindicating pupillary dilation.
- •Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including OCT, during the study period.
- •Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole, evident by ophthalmoscopy and/or by OCT examinations and assessed by the investigator to significantly affect central vision.
- •Current evidence of retinal detachment assessed by the investigator to significantly affect central vision.
- •Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
结局指标
主要结局
The safety and tolerability of escalating doses of vMCO-l administered via a single IVT in subjects with advanced Retinitis Pigmentosa
时间窗: 16 Weeks
Safety and tolerability of vMCO-l treatment at Week 16, by assessments based on local and systemic safety issues, as assessed by incidence of Adverse Events.
次要结局
- Evaluate the treatment effect of vMCO-l as assessed by visual acuity(52 weeks)
- Evaluate the treatment effect of vMCO-l as assessed by Quality of Life Questionnaire(52 weeks)
- Evaluate the treatment effect of vMCO-l as assessed by Humphrey Visual Field(52 weeks)
- Evaluate the treatment effect of vMCO-l as assessed by Visually-guided Mobility assays(52 weeks)
- Evaluate the treatment effect of vMCO-l as assessed by Static Shape recognition assay(52 weeks)
- Evaluate the treatment effect of vMCO-l as assessed by Optical Flow assay(52 weeks)
研究者
研究点 (2)
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