The trial is taking place at:

Martel Eye Medical Group | Rancho Cordova, CA

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A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration

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Status and phase

Phase 2


Age-Related Macular Degeneration


Drug: Placebo
Drug: Iptacopan (LNP023)

Study type


Funder types



2021-001797-31 (EudraCT Number)

Details and patient eligibility


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.

Full description

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.


146 estimated patients




50+ years old


No Healthy Volunteers

Inclusion criteria

  • 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 LNP023.
  • If not received previously, vaccination against Haemophilius influenzae infection should be given, if available and according to local regulations.

Exclusion criteria

  • 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.

Trial design

Primary purpose




Interventional model

Parallel Assignment


Triple Blind

146 participants in 2 patient groups, including a placebo group

Iptacopan (LNP023)
Experimental group
Iptacopan (LNP023) oral use capsules
Drug: Iptacopan (LNP023)
Placebo Comparator group
Placebo matched to study drug, oral use capsules
Drug: Placebo

Trial contacts and locations



Central trial contact

Novartis Pharmaceuticals; Novartis Pharmaceuticals

Data sourced from

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