A Phase 2 Randomized, Active-Controlled, Double-masked Trial of Intravitreal 4D-150 Gene Therapy in Adults With Diabetic Macular Edema (SPECTRA)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 72
- 试验地点
- 12
- 主要终点
- Incidence and severity of TEAEs and SAEs, including clinically significant changes in safety parameters (Part 1)
研究概览
简要总结
Phase 2 randomized, active-controlled, double-masked, dose-ranging trial in adults with Diabetic Macular Edema (DME).
详细描述
This Phase 2 trial is a prospective, multicenter, randomized, active-controlled, double-masked dose-ranging trial to evaluate the safety and efficacy of 4D-150 in adults with DME. The trial will be conducted in two parts: Dose Confirmation and Dose Expansion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Outcomes Assessor)
盲法说明
No masking in Dose Confirmation (Part 1). In Dose Expansion (Part 2), participants and outcomes assessors will be masked to the treatment arm for the duration of the trial.
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
- •≥18 years of age
- •Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea; diagnosis of DME must be within 2 years of Screening
- •CST ≥ 350 μm (Spectralis SD-OCT) in the study eye at Screening, confirmed by the independent reading center
- •Demonstrate clinical response to on-study aflibercept injection in the study eye.
- •Decreased visual acuity attributable primarily to DME
- •BCVA in the study eye between 25 and 83 ETDRS letters, inclusive (~20/320 and 20/25, respectively) at Screening
- •Study eye amenable to IVT injection
- •Sufficient clear ocular media, pupil dilation and fixation in the study eye to permit adequate imaging; ability to perform tests of visual and retinal function and structure; and ability to comply with other protocol-specified procedures
- •Provide written informed consent
排除标准
- •Macular edema in the study eye considered to be secondary to a cause other than DME
- •Systemic anti-VEGF treatment (e.g. sunitinib, bevacizumab, pazopanib) within 6 months, or anticipated need for systemic anti-VEGF therapy during study participation
- •Systemic corticosteroids (oral, intravenous, intramuscular, intra-articular) or other immunosuppressive medications within 3 months
- •Received an investigational drug, agent, device, or therapy (ocular or non-ocular) in the 3 months (or at least 5 half-lives, whichever is longer) prior to 4D-150 administration (Day 1)
- •Prior gene therapy (ocular or non-ocular) and/or ocular stem cell therapy in either eye
- •Any concurrent ocular condition in the study eye that is likely to require surgical intervention (e.g. cataract surgery) during the 2 year (104 week) study duration
- •Note: Other inclusion/exclusion criteria apply.
研究组 & 干预措施
4D-150 Part 2 Dose Expansion Dose Level 1
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
干预措施: 4D-150 IVT (Biological)
4D-150 Part 1 Dose Confirmation Dose Level 2
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
干预措施: 4D-150 IVT (Biological)
4D-150 Part 1 Dose Confirmation Dose Level 1
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
干预措施: 4D-150 IVT (Biological)
4D-150 Part 2 Dose Expansion Dose Level 2
4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day 1.
干预措施: 4D-150 IVT (Biological)
4D-150 Part 2 Dose Expansion Control
Aflibercept at a fixed regimen will be administered.
干预措施: Aflibercept IVT (Biological)
结局指标
主要结局
Incidence and severity of TEAEs and SAEs, including clinically significant changes in safety parameters (Part 1)
时间窗: 60 months
Annualized number of aflibercept injections in the study eye (Part 2)
时间窗: 52 weeks
次要结局
- Mean cumulative number of aflibercept injections over time(52 weeks)
- Change from baseline in BCVA as assessed using the ETDRS Visual Acuity Chart at Weeks 52 and 104(104 weeks)
- Mean cumulative number of aflibercept injections over time(52 weeks)
- Change from baseline in CST measured by spectral domain optical coherence tomography (SD-OCT) at Weeks 104(104 weeks)