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

• Status: Not yet recruiting
• Trial end date: 2026-09-08
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals

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:

• Concomitant medical or ocular conditions which could compromise visual acuity, require planned medical or surgical intervention during the study period, preclude scheduled study visits, completion of the study, or safe administration of the investigational product, including intraocular surgery, cataract and vitreoretinal surgery in the study eye within 3 months prior to Baseline/Day 1 and the presence of significant media opacity, eye movement disorder (nystagmus), severe ptosis, extraocular motility restriction or head tremor.

• History of clinically significant electrocardiogram (ECG) abnormalities, or any of the following ECG abnormalities at screening or Baseline/Day 1 visit:

• QT interval corrected by Fridericia's formula (QTcF) >450 msec (males)

• QTcF >460 msec (females)

• 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 kidney failure including end stage renal 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

• Chronic infection with Hepatitis B or Hepatitis C.

• 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.