A PHASE 1 STUDY TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE AND PHASE 2/3 PIVOTAL CONFIRMATORY CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- Ocugen
- 入组人数
- 51
- 试验地点
- 26
- 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
Phase 2/3 Pivotal Confirmatory Clinical Trial is a randomized, outcome assessor-masked, multicenter study, that will enroll fifty-one (51) subjects. Subjects will be enrolled in a 2:1 ratio to either the treatment group (n=34 subjects) or to an untreated control group (n=17 subjects).
Phase 1 is complete and closed for enrollment. It was a multicenter, open-label, dose ranging/dose escalation study that enrolled 9 subjects.
OCU410ST Phase 1- Retinal Structure and Visual Function Data Results
- Safety: Favorable safety and tolerability profile No SAE deemed related to OCU410ST including intraocular inflammation, exudation, endophthalmitis, anterior ischemic optic neuropathy (AIONs) or vasculitis.
- Primary Endpoint: Structural Improvement Atrophic lesions grew slower by 48% at 12M in evaluable treated eyes when compared to untreated eyes
- Secondary Endpoint: Visual Function (BCVA) 100% evaluable treated eyes demonstrated stabilization or improvement when compared to untreated eyes in visual function
OCU410ST Phase 1- Structural and Functional Outcomes at 12M Data Results
- The GARDian3 clinical trial for ABCA4- related retinopathies including Stargardt disease builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow-up in evaluable treated eyes compared to untreated eyes
- Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up when compared to untreated eyes.
详细描述
Name of Investigational Product: OCU410ST Name of Active Ingredient: Adeno-associated viral vector 5 human RORA (AAV5-hRORA)
Title of Study: A PHASE 1 STUDY TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE AND PHASE 2/3 PIVOTAL CONFIRMATORY CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE
Study Center(s): Approximately fifteen study centers in the US.
Background: Stargardt disease is an eye disease that causes vision loss in children and young adults. It is an inherited disease caused by faulty genes that cause buildup of fat deposits in the eye. Currently, there is no approved treatment available for Stargardt disease.
OCU410ST Product Information:
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
盲法说明
The masked biostatistician and other study team members identified by the masking plan
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Phase 2/3 Inclusion Criteria (applicable for both adult and pediatric subjects):
- •Males or females aged ≥5 years at the time of consent.
- •Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies).
- •Adult subjects who have BCVA of 75 letters or less in the study eye (20/32 Snellen equivalent) and Pediatric subjects who have a BCVA of 35 letters or better in the study eye (20/200 Snellen equivalent).
- •The study eye should have at least one well-demarcated area of atrophy with a minimum diameter of 300 microns, and total lesion size must add to less than or equal to ≤ 18 mm^2 (approximately 7-disc areas).
- •Have detectable outer nuclear layer (ONL) in the macular region
排除标准
- •(applicable for both adult and pediatric subjects):
- •Participation in ongoing antiretroviral therapy treatment.
- •Participation in any investigational therapy study or receipt of investigational treatment within 60 days prior to screening or 5 half-lives (whichever is longer).
- •Any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, intravitreal or subretinal injections, or participation in an Alkeus ALK-001 study within the past 6 months.
- •Macular atrophy secondary to any disease other than Stargardt Disease (STGD).
- •Presence of genetic mutations that mimic Stargardt Disease like ELOVL4, or PROM
- •Contraindication to subretinal injection or use of anesthesia (local and/or general).
- •Phase 1 was a multicenter, open-label, dose-ranging/dose escalation study. Enrollment is complete for Phase 1
研究组 & 干预措施
Phase 2/3 Randomized Treatment Arm
Subjects will receive a single subretinal injection of 200uL OCU410ST in concentration of 1.5 x 10E11vg/mL
干预措施: OCU410ST (Drug)
Phase 2/3 Randomized Control Arm
Subjects will not receive any active study intervention
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: 12 months (Screening to 12 months post OCU410ST administration)
Safety will be 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).
Change from baseline in atrophic lesion size as measured by Fundus Auto Fluorescence
时间窗: 12 months (Screening to 12 months post OCU410ST administration)
Change in the area of atrophy will be evaluated from the baseline measurements, using FAF to assess the loss of retinal layers.
Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
时间窗: 12 months (Screening to 12 months post OCU410ST administration)
Safety will be 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
- Change from baseline in Best Corrected Visual Acuity (BCVA)(12 months (Screening to 12 months post OCU410ST administration))
- Change from baseline in Low Luminance Visual Acuity (LLVA)(12 months (Screening to 12 months post OCU410ST administration))