A Phase 1/2 Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, and Efficacy of One-time Intravitreal Dose of SAR402663 in Participants With Neovascular Age-related Macular Degeneration
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Sanofi
- 入组人数
- 66
- 试验地点
- 44
- 主要终点
- Incidence and severity of ocular treatment emergent adverse event (TEAEs)
研究概览
简要总结
This is a Phase 1/Phase 2 multicenter study to evaluate the safety and efficacy of a one-time single-eye intravitreal dose of SAR402663 in participants with neovascular age-related macular degeneration.
Participants will be enrolled in one of 2 parts:
- In Part I (dose escalation), multiple dose levels of SAR402663 will be evaluated in successive cohorts of participants
- In Part II (dose expansion), participants will be randomized to receive one of two dose levels selected based on data from Part I. Participants, investigators and outcomes assessors will be masked to dose.
After receiving one-time dose of SAR402663, participants will undergo regular assessments over 12 months. Following this, participants will enter an extended follow-up (EFU) phase for the assessment of safety and durability of clinical activity of SAR402663 through Year 5.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Part I is an open-label study. Part II is partially masked in which participants, Investigators, and outcomes assessors including reading center (RC) are masked to study intervention. The Sponsor is not masked. Masking will be maintained until all participants reach 52 weeks of follow up after SAR402663 dose.
入排标准
- 年龄范围
- 50 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Between 50 and 90 years of age
- •Participants with diagnosis of macular neovascularization secondary to age-related macular degeneration (nAMD)
- •Study eye with best corrected visual acuity (BCVA) ETDRS Snellen equivalent for dose escalation (Part I) between 20/32 and 20/400 and for expansion (Part II) between 20/25 and 20/200
- •Current or previous use of anti-vascular endothelial growth factor (VEGF) treatment in the study eye
- •Demonstrated a response to anti-VEGF treatment
排除标准
- •Any condition in the study eye that may prevent visual acuity improvement or interfere with ocular safety or efficacy assessments
- •History of active ocular infection in the study eye in 6 months prior to screening
- •Active uncontrolled glaucoma in the study eye
- •History of uveitis in either eye
- •Current use of ocular corticosteroids in the study eye
- •Previous gene therapy
- •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 potential participation in a clinical trial.
研究组 & 干预措施
Part I - SAR402663 open-label (OL)
Participants will receive a single dose of SAR402663 on Day 1. Multiple dose levels of SAR402663 will be evaluated in successive cohorts of participants.
干预措施: SAR402663 (Biological)
Part II - SAR402663 Dose A
Participants will receive a single dose of SAR402663 on Day 1.
干预措施: SAR402663 (Biological)
Part II - SAR402663 Dose B
Participants will receive a single dose of SAR402663 on Day 1.
干预措施: SAR402663 (Biological)
Part I - SAR402663 open-label (OL)
Participants will receive a single dose of SAR402663 on Day 1. Multiple dose levels of SAR402663 will be evaluated in successive cohorts of participants.
干预措施: Diluent (Drug)
Part II - SAR402663 Dose A
Participants will receive a single dose of SAR402663 on Day 1.
干预措施: Diluent (Drug)
Part II - SAR402663 Dose B
Participants will receive a single dose of SAR402663 on Day 1.
干预措施: Diluent (Drug)
结局指标
主要结局
Incidence and severity of ocular treatment emergent adverse event (TEAEs)
时间窗: Day 1 to Week 52
Incidence and severity of ocular treatment emergent serious adverse event (TESAEs)
时间窗: Day 1 to Week 52
Incidence and severity of non-ocular TEAEs
时间窗: Day 1 to Week 52
Incidence and severity of non-ocular TESAEs
时间窗: Day 1 to Week 52
Number of participants with any clinically significant changes in laboratory variables
时间窗: Day 1 to Week 52
Number of participants with any clinically significant changes in vital signs
时间窗: Day 1 to Week 52
次要结局
- Annualized injection rates of anti-VEGF therapy(Day 1 to Week 52)
- Change from baseline in BCVA using the ETDRS letter score(Baseline, Week 52)
- Percentage of participants not losing more than or equal to 15 ETDRS letters from baseline(Baseline, Week 52)
- Change from baseline in central subfield thickness (CST), measured by spectral domain optical coherence tomography (SD-OCT)(Baseline, Week 52)
- Percentage of participants not requiring supplemental anti- vascular endothelial growth factor (VEGF) therapy(Day 1 to Week 52 and Week 8 to Week 52)
- Percentage of participants not requiring supplemental anti-vascular endothelial growth factor (VEGF) therapy(Day 1 to Week 52 and Week 8 to Week 52)
- Annualized injection rates of anti-VEGF therapy(Day 1 to Week 52)
- Change from baseline in BCVA using the ETDRS letter score(Baseline, Week 52)
- Percentage of participants not losing more than or equal to 15 ETDRS letters from baseline(Baseline, Week 52)
- Change from baseline in central subfield thickness (CST), measured by spectral domain optical coherence tomography (SD-OCT)(Baseline, Week 52)