A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- Ocugen
- 入组人数
- 140
- 试验地点
- 27
- 主要终点
- Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control
研究概览
简要总结
This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP.
This is a multicenter, assessor blinded and randomized study which will enroll 140 subjects. Study has completed enrollment of all 140 subjects.
详细描述
A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.
Subjects in each arm will be randomized into treatment and control groups with a 2:1 ratio. Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.
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
- Single (Outcomes Assessor)
盲法说明
This is outcome assessor blinded study
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females ≥ 3 years of age
- •Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
- •Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
- •BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart
- •Visual field of >5° in any meridian as measured by a III4e isopter or equivalent
- •Able to perform a Luminance LDNA at certain light intensity at the Screening visit
- •Presence of photoreceptors as determined by SD-OCT
排除标准
- •Subject lacks evidence of outer nuclear layer
- •Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
- •History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
- •Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
- •Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
- •Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception
研究组 & 干预措施
Control for RHO Arm
Will not receive any active study intervention
Control for Gene Agnostic Arm
Will not receive any active study intervention
RHO Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye
干预措施: Sub-Retinal Administration of OCU400-301 (Genetic)
Gene Agnostic Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10^10 vg/eye
干预措施: Sub-Retinal Administration of OCU400-301 (Genetic)
结局指标
主要结局
Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control
时间窗: 52 weeks
Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
次要结局
- Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores(52 weeks)
- Change in functional vision from baseline to week 52 in all the treated eyes from RP subjects (study eyes + fellow treated eyes) when compared to all the eyes (study eyes and fellow eyes) in untreated control group(52 weeks)
- Change from Baseline in visual function in patients with retinitis pigmentosa when treatment group were compared to untreated controls(52 weeks)
- Change from baseline in LDNA Lux-level results in treated subjects when compared to the untreated controls(52 weeks)