Phase 1, Open-Label, Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-015 in Patients With Retinitis Pigmentosa or Choroideremia (ENVISION)
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
- 4
- 主要终点
- Incidence of Treatment-Emergent Adverse Events
研究概览
简要总结
A Phase 1, open-label, non-randomized, dose-escalation study, where approximately 18 eligible patients with retinitis pigmentosa or choroideremia will be enrolled sequentially in up to 4 dose cohorts of RTx-015. Enrolled patients will receive a single, unilateral intravitreal injection of RTx-015 in the study eye at Visit 3 (Day 0) and be followed for a total of 5 years.
详细描述
This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-015, in patients with retinitis pigmentosa or choroideremia. Up to 4 dose cohorts are planned, and each cohort will consist initially of 3 patients. Eligible patients will be assigned to a dose cohort by sequential enrollment.
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
- •Adults ≥18 years of age
- •Informed consent obtained from the patient
- •Clinical diagnosis of Retinitis Pigmentosa independent of causative mutation OR Clinical diagnosis of Choroideremia with known causative mutation
- •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, in the opinion of the Investigator, 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; complicating systemic diseases include those in which the disease itself, or the treatment for the disease, can alter ocular and/or Central Nervous System (CNS) function (e.g., radiation treatment of the orbit; leukemia with CNS/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-015
Single intravitreal injection of low dose RTx-015
干预措施: RTx-015 (Genetic)
Middle Dose RTx-015
Single intravitreal injection of middle dose RTx-015
干预措施: RTx-015 (Genetic)
High Dose RTx-015
Single intravitreal injection of high dose RTx-015
干预措施: RTx-015 (Genetic)
Higher Dose RTx-015
Single intravitreal injection of higher dose RTx-015
干预措施: RTx-015 (Genetic)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events
时间窗: 12 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) at Month 6 and Month 12(6 and 12 Months)
- Contrast Sensitivity at Month 6 and Month 12(6 and 12 Months)
- Low Vision Quality of Life at Month 6 and Month 12(6 and 12 Months)
- Low Luminance Visual Acuity (LLVA) at Month 6 and Month 12(6 and 12 Months)
- Multi luminance mobility at Month 6 and Month 12(6 and 12 Months)
- Full-field static visual field testing at Month 6 and Month 12(6 and 12 Months)