A Masked, Placebo-controlled Study to Assess Iptacopan in 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
2 other identifiers
interventional
170
4 countries
28
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Feb 2022
Typical duration for phase_2
28 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 28, 2022
CompletedFirst Posted
Study publicly available on registry
February 9, 2022
CompletedStudy Start
First participant enrolled
February 17, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 2, 2026
July 3, 2025
July 1, 2025
4.6 years
January 28, 2022
July 2, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
OCT and other imaging will be performed using spectral domain OCT or swept source OCT machines
Baseline/Day 1 through Month 24
Secondary Outcomes (7)
The incidence of ocular and non-ocular adverse events (AEs)
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 contrast sensitivity (CS) scores in the early/intermediate AMD eye
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
- +2 more secondary outcomes
Study Arms (2)
Iptacopan (LNP023)
EXPERIMENTALIptacopan (LNP023) oral use capsules
Placebo
PLACEBO COMPARATORPlacebo matched to study drug, oral use capsules
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (28)
Retina Consultants of Orange County
Fullerton, California, 92835, United States
Salehi Retina Institute
Huntington Beach, California, 92647, United States
Martel Eye Medical Group
Rancho Cordova, California, 95670, United States
Retinal Consultants Medical Group Inc
Sacramento, California, 95841, United States
California Retina Consultants
Santa Barbara, California, 93103, United States
Southwest Retina Research Center
Durango, Colorado, 81303, United States
Advanced Research LLC
Deerfield Beach, Florida, 33064, United States
Retina Center Of South Florida
Delray Beach, Florida, 33484, United States
Retina Vitreous Associates of Florida
St. Petersburg, Florida, 33711, United States
Midwest Eye Institute
Indianapolis, Indiana, 46280, United States
Retina Associates New Orleans
New Orleans, Louisiana, 70115 8139, United States
Retina Care Center
Baltimore, Maryland, 21209, United States
Opthamalic Consultants of Boston
Boston, Massachusetts, 02114, United States
Envision Ocular LLC
Bloomfield, New Jersey, 07003, United States
Duke Eye Center
Durham, North Carolina, 27705, United States
Retina Northwest PC
Portland, Oregon, 97210, United States
Charles Retina Institute
Germantown, Tennessee, 38138, United States
Austin Research Center for Retina
Austin, Texas, 78793, United States
Retina Consultants TX Rsrch Ctr
Bellaire, Texas, 77401, United States
Retina Foundation
Dallas, Texas, 75231, United States
Texas Retina Associates
Fort Worth, Texas, 76104, United States
Retina Consultants of Houston PA
Houston, Texas, 77030, United States
Novartis Investigative Site
Harbin, Heilongjiang, 150001, China
Novartis Investigative Site
Tianjin, Tianjin Municipality, 300020, China
Novartis Investigative Site
Shanghai, 200080, China
Emanuelli Research and Development Center LLC
Arecibo, 00612, Puerto Rico
Novartis Investigative Site
London, SE5 9RS, United Kingdom
Novartis Investigative Site
Southampton, SO16 6YD, United Kingdom
Related Publications (1)
Tzoumas N, Riding G, Williams MA, Steel DH. Complement inhibitors for age-related macular degeneration. Cochrane Database Syst Rev. 2023 Jun 14;6(6):CD009300. doi: 10.1002/14651858.CD009300.pub3.
PMID: 37314061DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Masking Details
- Investigator and Participant
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 28, 2022
First Posted
February 9, 2022
Study Start
February 17, 2022
Primary Completion (Estimated)
October 1, 2026
Study Completion (Estimated)
October 2, 2026
Last Updated
July 3, 2025
Record last verified: 2025-07
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.