A Phase 1/2, Study to Evaluate the Safety, Tolerability, and Efficacy of One-time Intravitreal Dose of SAR446597 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- Sanofi
- 入组人数
- 104
- 试验地点
- 24
- 主要终点
- Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs)
研究概览
简要总结
This is a sequential Phase 1/2, two-part, multicenter study on safety, tolerability, and efficacy of one-time intravitreal SAR446597 for the treatment of participants with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD).
The core phase duration will be approximately 2 years for each participant. An Extended Follow-Up (EFU) phase of 3 years follows the core phase.
The treatment is a one-time intravitreal injection of SAR446597 (or sham as applicable in Part II).
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
- Double (Participant, Outcomes Assessor)
入排标准
- 年龄范围
- 60 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •60 years old or above
- •Participants with diagnosis of GA secondary to age-related macular degeneration (AMD)
- •Study eye with best corrected visual acuity (BCVA) ETDRS Snellen equivalent for dose escalation (Part I) between 20/40 and 20/320 and for expansion (Part II) equal or better than 20/200
- •Study eye with GA lesion measuring between 2.5 and 17.5 mm2 for dose escalation (Part I) and between 2.5 and 14.0 mm2 for expansion (Part II). For multifocal disease, study eye with at least one single lesion of more than 1.25mm2 for both Part I and Part II
排除标准
- •GA in the study eye caused by a disease different than AMD
- •Presence of neovascularization or a history of treatment with an anti vascular endothelial growth factor agent in the study eye
- •Any condition or treatment (ocular or systemic) or medical or surgical history in the study eye that may prevent visual acuity improvement or interfere with ocular safety or efficacy assessments
- •Current or history of systemic complement targeting treatment in the past 12 months
- •Use of ocular corticosteroids for 4 months (for ocular or periocular injections), 6 months (for intraocular implants) or 3 years (for long lasting intraocular implants) prior to screening in the study eye
- •History of macular laser photocoagulation treatment, photodynamic- or thermotherapy or photo biomodulation in the study eye
- •History of active ocular infection in the study eye in 6 months prior to screening
- •Presence of active ocular or periocular infections
- •Active uncontrolled glaucoma in the study eye
- •History of uveitis or scleritis in either eye
- •Previous gene therapy in either eye
- •Any significant poorly controlled illness that would preclude study compliance and follow up
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
研究组 & 干预措施
Part I - SAR446597 open-label (OL)
Participants will receive SAR at a dose specified for each cohort. Multiple dose levels of SAR446597 will be evaluated in successive cohorts of participants.
干预措施: SAR446597 (Drug)
Part II - SAR446597 Dose A
Participants will receive SAR at a dose specified for each arm.
干预措施: SAR446597 (Drug)
Part II - SAR446597 Dose B
Participants will receive SAR at a dose specified for each arm.
干预措施: SAR446597 (Drug)
Part II - Sham control
Participants will receive Sham at a dose specified for each arm.
干预措施: Sham Comparator (Drug)
结局指标
主要结局
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs)
时间窗: Day 1 to Week 104
Incidence and severity of ocular and non-ocular treatment-emergent serious adverse events (TESAEs)
时间窗: Day 1 to Week 104
次要结局
- Change in square root-transformed (mm) and untransformed area (mm2) of GA(Baseline, Week 52, Week 104)
- Change in best-corrected visual acuity (BCVA) from baseline to Week 52 and Week 104 following SAR446597 administration measured with the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart(Baseline, Week 52, Week 104)
- Percentage of participants without loss of BCVA of ≥15 ETDRS letters from baseline in the study eye(Baseline, Week 52, Week 104)
- Incidence and severity of ocular and non-ocular TEAEs and TESAEs including clinically significant changes in safety parameters(Week 260 or End of Study)