A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 10
- 试验地点
- 3
- 主要终点
- The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa
研究概览
简要总结
The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection and repeated light stimulation using a medical device called GS030-MD (stimulating glasses) in subjects with documented diagnosis of non-syndromic Retinitis Pigmentosa
详细描述
Study GS030_CLIN_001 is a multicenter, Phase 1/2a, open-label, non-randomized, dose-escalation, safety and tolerability study of GS030-DP in association with GS030-MD in subjects with non-syndromic RP that is confirmed by full-field ERG. The study design includes a dose escalation of the vector encoding the ChR-tdT. This first-in-human study design evaluates the safety and tolerability of GS030, the associated GS030-MD and GS030-DP, which is the treatment to be developed and considered for marketing.
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 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years to ≤75 years at the time of ICF signature.
- •Diagnosis of non-syndromic RP defined as:
- •Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance.
- •Diagnosis of non-syndromic RP is confirmed on full-field ERG
- •Visual acuity:
- •Visual acuity in the dose-escalation cohorts of no better LP.
- •Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB.
- •Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT).
- •Interpupillary distance of ≥51 mm and ≤72 mm.
- •Refractive error of the study eye between -6 diopters and +6 diopters.
排除标准
- •Prior receipt of any gene therapy.
- •Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit
- •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 SD-OCT, during the study period.
- •Presence of any systemic or ocular diseases, or pathologies, other than non-syndromic RP, or their associated therapies, that can cause or have the potential to cause vision loss.
- •Prior vitrectomy or vitreomacular surgery.
- •Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole, evident by ophthalmoscopy and/or by SD-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.
- •Presence of an Active Implantable Medical Device.
- •Subjects who have undergone thermal laser procedure to the retina within 3 months of trial entry, or any prior thermal laser procedure to the macular region.
研究组 & 干预措施
Cohort
3 dose escalation cohorts (low, medium and high dose) with 3 subjects per cohort followed by an extension cohort at the highest-well tolerated dose with 3 to 9 subjects.
干预措施: Gene therapy: GS030-DP AND Medical device: GS030-MD (Combination Product)
结局指标
主要结局
The safety and tolerability of escalating doses of GS030-DP administered via a single IVT and repeated light stimulation using GS030-MD in subjects with non-syndromic Retinitis Pigmentosa
时间窗: Week 52/Year 1
Safety and tolerability of GS030 treatment at Week 52/Year 1, by assessments based on local and systemic safety issues, specifically those related to IVT of GS030-DP and the subsequent repeated use of GS030-MD, as assessed by incidence of Adverse Events.
次要结局
- Evaluate the treatment effect of GS030 as assessed by visual acuity(Week 52/Year 1)
- Evaluate the treatment effect of GS030 as assessed by mobility (line task).(Week 52/Year 1)
- Evaluate the treatment effect of GS030 as assessed by QoL (SF36)(Week 52/Year 1)
- Evaluate the treatment effect of GS030 as assessed by visual function(Week 52/Year 1)
- Evaluate the treatment effect of GS030 as assessed by mobility (door task).(Week 52/Year 1)
- Evaluate the treatment effect of GS030 as assessed by QoL (VFQ25)(Week 52/Year 1)
- Evaluate immune response to recombinant adeno associated viral vector, derived from serotype 2 (rAAV2.7m8) and ChR tdT protein.(Week 52/Year 1)