Phase 1/2, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease (AURORA)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 3
- 主要终点
- Incidence of Treatment-Emergent Adverse Events
研究概览
简要总结
A Phase 1/2, open-label, non-randomized, dose-escalation study, where a minimum of 9 and a maximum of 18 eligible patients with Stargardt Disease will be enrolled sequentially in up to 3 dose cohorts of RTx-021. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years.
详细描述
This Phase 1/2, open-label, non-randomized, sequential assignment, first-in-human dose-escalation study is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-021, in patients with Stargardt Disease. Up to 3 dose cohorts of RTx-021 are planned, and each cohort will consist initially of 3 patients. The study is a traditional dose escalation design with the potential for cohort expansion and is intended to support dose selection for further clinical development. Patients will receive a single, unilateral intravitreal injection of RTx-021 in the study eye and be followed for 5 years with visits being more frequent in the initial 12 months after treatment.
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
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients >= 16 years of age
- •Able to comply with the study visit schedule and all protocol assessments
- •Diagnosis of Stargardt Disease (genetic testing required)
- •Study eye and fellow eye Best Corrected Visual Acuity meeting study criteria
- •Presence of retinal ganglion cells and/or retinal nerve fiber layer on Spectral Domain Optical Coherence Tomography testing at Screening confirmed by central image reading center
- •Adequate organ function and general good health
排除标准
- •Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent, or therapy in the past six months
- •Concurrent participation in another interventional clinical ocular study
- •Prior receipt of any gene therapy (ocular or other), retinal implant, or ocular cell therapy
- •Pre-existing eye conditions in either eye that would preclude the planned treatment or are significant enough to interfere with the interpretation of study endpoints or procedural complications
- •Known steroid responders if their intraocular pressure was not able to be managed effectively with topical pressure-lowering medications after prior use of steroid medications
- •Complicating systemic diseases including those in which the disease itself, or the treatment of the disease, can alter ocular and/or central nervous system function (e.g. radiation treatment of the orbit; leukemia with optic nerve involvement)
- •Any immunological response dysfunction including, immuno-compromising diseases or use of immunosuppressive medications, among others
- •Cataract or other ocular (including refractive) surgery, intraocular and/or peri-ocular injection in either eye within the prior four months (i.e. 120 days) prior to screening
- •Prior vitrectomy or aphakia in the study eye
- •Known sensitivity to any component of the study treatment or contraindication to medications planned for use in the peri-procedural period (e.g. povidone-iodine to prep for intravitreal injection)
- •Known contraindication to prophylactic steroid regimen
- •Current pregnancy or breastfeeding
- •Any other condition that would not allow the patient to complete follow-up examinations during the study
研究组 & 干预措施
Low Dose RTx-021
Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.
干预措施: RTx-021 (Genetic)
Middle Dose RTx-021
Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.
干预措施: RTx-021 (Genetic)
High Dose RTx-021
Intravitreal injection using gene therapy to deliver an optogenetic gene to the eye. Cells in the retina use this optogenetic gene to make a protein that responds to light.
干预措施: RTx-021 (Genetic)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events
时间窗: 6 Months
The number of patients in each cohort with treatment-emergent adverse events categorized using MedDRA v24.0 or higher
次要结局
- Best Corrected Visual Acuity (BCVA)(6 Months)
- Low Luminance Visual Acuity (LLVA)(6 Months)
- MNREAD Reading Assessment(6 Months)
- Contrast Sensitivity(6 Months)
- Visual Field(6 Months)
- Low Vision Quality of Life (VA LV VFQ-48)(6 Months)