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Assessing Iptacopan for Age-related Macular Degeneration

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

Phase 2 Interventional Novartis · NCT05230537

This study is testing if a new drug called Iptacopan can help prevent early or intermediate age-related macular degeneration from getting worse in people who have it in one eye and a more severe form in the other.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment146 (estimated)
Ages50 Years and up
SexAll
SponsorNovartis Industry-sponsored
Locations32 sites (Fullerton, California and 31 other locations)
Trial IDNCT05230537 on ClinicalTrials.gov

What this trial studies

This study evaluates the effectiveness of Iptacopan (LNP023) in preventing the progression of early or intermediate age-related macular degeneration (AMD) to more severe forms. It is a multicenter, randomized, participant and investigator masked, placebo-controlled trial involving participants with AMD in one eye and neovascular AMD in the other. Approximately 146 participants will be randomly assigned to receive either Iptacopan or a placebo, with the aim of determining the safety and efficacy of the treatment.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 50 and older with early or intermediate AMD in one eye and neovascular AMD in the other eye.

Not a fit: Patients with significant cardiac arrhythmias or other serious health conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could help preserve vision and prevent the progression of AMD in affected patients.

How similar studies have performed: Other studies have shown promise in treating AMD, but this specific approach with Iptacopan is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Fullerton, California and 31 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Age-Related Macular DegenerationAge-related macular degenerationMacular degenerationvision loss, macula damageretina damagedry macular degenerationwet macular degenerationAMD
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.