A Randomized, Participant and Investigator Masked, Placebo-controlled, Multicenter, Proof-of-concept Study to Assess the Safety and Efficacy of LNP023 (Iptacopan) in Patients With Early and Intermediate Age-related Macular Degeneration
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 170
- 试验地点
- 24
- 主要终点
- Development of new incomplete retinal pigment epithelium & outer retinal atrophy or late age-related macular degeneration (AMD) in the early/intermediate AMD eye as determined by optical coherence tomography (OCT) & supported by multimodal imaging
研究概览
简要总结
The purpose of this study is to assess the effect of Iptacopan to prevent conversion of early or intermediate age-related macular degeneration (AMD) eyes to new incomplete retinal pigment epithelium and outer retinal atrophy (iRORA) or late AMD.
详细描述
This is a multicenter, randomized, participant and investigator masked, placebo controlled, proof-of-concept study to assess the safety and efficacy of Iptacopan (LNP023) in participants with early to intermediate age-related macular degeneration in one eye and neovascular age-related macular degeneration in the other eye. All enrolled participants must have early/intermediate AMD in one eye, with at least one high risk optical coherence tomography (OCT) feature (study eye) and neovascular AMD in the other eye (fellow eye).
Participants who meet all of the eligibility criteria will be randomized at the Baseline/Day 1 visit in a 1:1 ratio into one of two treatment arms:
- Iptacopan (LNP023) oral capsules
- Placebo oral capsules Approximately 146 participants (73 per arm) will be treated worldwide.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
盲法说明
Investigator and Participant
入排标准
- 年龄范围
- 50 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants ≥ 50 years of age
- •Diagnosis of early or intermediate age-related macular degeneration (AMD) in the study eye as determined by the investigator on fundus examination
- •Study eye (early/intermediate AMD eye) must have at least one high risk optical coherence tomography (OCT) feature (as defined by a central reading center).
- •Diagnosis of neovascular AMD (nAMD) in the fellow eye as determined by the investigator.
- •Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection are required prior to the start of the treatment with LNP
- •If not received previously, vaccination against Haemophilius influenzae infection should be given, if available and according to local regulations.
排除标准
- •History or current diagnosis of ECG abnormalities indicating significant safety risk, such as clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia and clinically significant second or third degree atrioventricular block (AV block) without a pacemaker.
- •History of familial long QT syndrome or known family history of Torsades de Pointes
- •History of stroke or myocardial infarction during the 6-month period prior to Baseline/Day 1, any current clinically significant arrhythmias, or any advanced cardiac or severe pulmonary hypertension
- •History of end stage kidney disease requiring dialysis or renal transplant
- •History of malignancy of any organ system
- •History of solid organ or bone marrow transplantation
- •History of recurrent meningitis or history of meningococcal infections despite vaccination
- •History of immunodeficiency diseases, including a positive Human Immunodeficiency Virus test result at Screening
- •Active Hepatitis B (HBV) or Hepatitis C (HCV) infection
- •History of hypersensitivity to any of the study treatments or excipients or to drugs of similar chemical classes or clinically relevant sensitivity to fluorescein dye as assessed by the Investigator.
- •Evidence of cRORA or exMNV in the study eye based on multimodal imaging as determined by the central reading center.
- •Participants who have current active TB as evidenced by clinical, radiographic and laboratory tests.
研究组 & 干预措施
Iptacopan (LNP023)
Iptacopan (LNP023) oral use capsules
干预措施: Iptacopan (LNP023) (Drug)
Placebo
Placebo matched to study drug, oral use capsules
干预措施: Placebo (Drug)
结局指标
主要结局
Development of new incomplete retinal pigment epithelium & outer retinal atrophy or late age-related macular degeneration (AMD) in the early/intermediate AMD eye as determined by optical coherence tomography (OCT) & supported by multimodal imaging
时间窗: Baseline/Day 1 through Month 24
OCT and other imaging will be performed using spectral domain OCT or swept source OCT machines
次要结局
- Change in contrast sensitivity (CS) scores in the early/intermediate AMD eye(Baseline/Day 1 through Month 24)
- The incidence of ocular and non-ocular adverse events (AEs)(Baseline/Day 1 through Month 24)
- Change in Standard luminance manifold contrast vision meter contrast sensitivity (MCVM-CS) scores in the early/intermediate AMD eye(Baseline/Day 1 through Month 24)
- Change in Early Treatment Diabetic Retinopathy Study (ETDRS) (Standard Luminance) best corrected visual acuity (BCVA) scores in the early/intermediate AMD eye(Baseline/Day 1 through Month 24)
- Change in ETDRS low luminance visual acuity (LLVA) scores in the early/intermediate AMD eye(Baseline/Day 1 through Month 24)
- Change in low luminance manifold contrast vision meter (MCVM) contrast sensitivity scores in the early/intermediate AMD eye(Baseline/Day 1 through Month 24)
- Pharmacokinetics - concentrations of LNP023 related to trough samples(Baseline/Day 1 through Month 24)