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

Last updated: July 2, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

2

Condition

Aging

Macular Degeneration

Geographic Atrophy

Treatment

Iptacopan (LNP023)

Placebo

Clinical Study ID

NCT05230537
CLNP023E12201
2021-001797-31
  • Ages 50-100
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Male or female participants ≥ 50 years of age

  • Diagnosis of early or intermediate age-related macular degeneration (AMD) in thestudy eye as determined by the investigator on fundus examination

  • Study eye (early/intermediate AMD eye) must have at least one high risk opticalcoherence tomography (OCT) feature (as defined by a central reading center).

  • Diagnosis of neovascular AMD (nAMD) in the fellow eye as determined by theinvestigator.

  • Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infectionare required prior to the start of the treatment with LNP023.

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

Exclusion

Exclusion Criteria:

  • History or current diagnosis of ECG abnormalities indicating significant safetyrisk, such as clinically significant cardiac arrhythmias, e.g., sustainedventricular tachycardia and clinically significant second or third degreeatrioventricular 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 toBaseline/Day 1, any current clinically significant arrhythmias, or any advancedcardiac 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 despitevaccination

  • History of immunodeficiency diseases, including a positive Human ImmunodeficiencyVirus 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 drugsof similar chemical classes or clinically relevant sensitivity to fluorescein dye asassessed by the Investigator.

  • Evidence of cRORA or exMNV in the study eye based on multimodal imaging asdetermined by the central reading center.

  • Participants who have current active TB as evidenced by clinical, radiographic andlaboratory tests.

Study Design

Total Participants: 170
Treatment Group(s): 2
Primary Treatment: Iptacopan (LNP023)
Phase: 2
Study Start date:
February 17, 2022
Estimated Completion Date:
October 02, 2026

Study 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.

Connect with a study center

  • Novartis Investigative Site

    Harbin, Heilongjiang 150001
    China

    Site Not Available

  • Novartis Investigative Site

    Harbin City, Heilongjiang 150000
    China

    Site Not Available

  • Novartis Investigative Site

    Tianjin, Tianjin 300020
    China

    Site Not Available

  • Novartis Investigative Site

    Shanghai, 200080
    China

    Site Not Available

  • Emanuelli Research and Development Center LLC

    Arecibo, 00612
    Puerto Rico

    Site Not Available

  • Novartis Investigative Site

    Arecibo, 00612
    Puerto Rico

    Active - Recruiting

  • Novartis Investigative Site

    Gloucester, GL1 3NN
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    London, SE5 9RS
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Southampton, SO16 6YD
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Fullerton, California 92835
    United States

    Active - Recruiting

  • Retina Consultants of Orange County

    Fullerton, California 92835
    United States

    Site Not Available

  • Novartis Investigative Site

    Huntington Beach, California 92647
    United States

    Active - Recruiting

  • Salehi Retina Institute

    Huntington Beach, California 92647
    United States

    Site Not Available

  • Martel Eye Medical Group

    Rancho Cordova, California 95670
    United States

    Site Not Available

  • Novartis Investigative Site

    Rancho Cordova, California 95670
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Sacramento, California 95841
    United States

    Active - Recruiting

  • Retinal Consultants Medical Group Inc

    Sacramento, California 95841
    United States

    Site Not Available

  • Retinal Consultants Medical Group, Inc

    Sacramento, California 95841
    United States

    Site Not Available

  • California Retina Consultants

    Santa Barbara, California 93103
    United States

    Site Not Available

  • California Retina Consultants .

    Santa Barbara, California 93103
    United States

    Site Not Available

  • Novartis Investigative Site

    Durango, Colorado 81303
    United States

    Active - Recruiting

  • Southwest Retina Research Center

    Durango, Colorado 81303
    United States

    Site Not Available

  • Advanced Research LLC

    Coral Springs, Florida 33067
    United States

    Site Not Available

  • Novartis Investigative Site

    Coral Springs, Florida 33067
    United States

    Active - Recruiting

  • Advanced Research LLC

    Deerfield Beach, Florida 33064
    United States

    Site Not Available

  • Advanced Research LLC Research

    Deerfield Beach, Florida 33064
    United States

    Site Not Available

  • Novartis Investigative Site

    Delray Beach, Florida 33484
    United States

    Active - Recruiting

  • Retina Center Of South Florida

    Delray Beach, Florida 33484
    United States

    Site Not Available

  • Novartis Investigative Site

    Saint Petersburg, Florida 33711
    United States

    Active - Recruiting

  • Retina Vitreous Associates of Florida

    Saint Petersburg, Florida 33711
    United States

    Site Not Available

  • Midwest Eye Institute

    Indianapolis, Indiana 46280
    United States

    Site Not Available

  • Midwest Eye Institute Research

    Indianapolis, Indiana 46280
    United States

    Site Not Available

  • Novartis Investigative Site

    Indianapolis, Indiana 46280
    United States

    Active - Recruiting

  • Novartis Investigative Site

    New Orleans, Louisiana 70115-8139
    United States

    Active - Recruiting

  • Retina Associates New Orleans

    New Orleans, Louisiana 70115 8139
    United States

    Site Not Available

  • Retina Associates New Orleans .

    New Orleans, Louisiana 70115 8139
    United States

    Site Not Available

  • Retina Care Center

    Baltimore, Maryland 21209
    United States

    Site Not Available

  • Retina Care Center Research

    Baltimore, Maryland 21209
    United States

    Site Not Available

  • Novartis Investigative Site

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

  • Opthamalic Consultants of Boston

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Envision Ocular LLC

    Bloomfield, New Jersey 07003
    United States

    Site Not Available

  • Vitreous Retina Macula Consultants Research

    New York, New York 10022
    United States

    Site Not Available

  • Duke Eye Center

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Duke Eye Center Research

    Durham, North Carolina 27705
    United States

    Site Not Available

  • Novartis Investigative Site

    Portland, Oregon 97210
    United States

    Active - Recruiting

  • Retina Northwest PC

    Portland, Oregon 97210
    United States

    Site Not Available

  • Retina Northwest PC .

    Portland, Oregon 97210
    United States

    Site Not Available

  • Novartis Investigative Site

    Kingston, Pennsylvania 18704
    United States

    Site Not Available

  • Charles Retina Institute

    Germantown, Tennessee 38138
    United States

    Site Not Available

  • Novartis Investigative Site

    Germantown, Tennessee 38138
    United States

    Active - Recruiting

  • Austin Research Center for Retina

    Austin, Texas 78793
    United States

    Site Not Available

  • Austin Research Center for Retina .

    Austin, Texas 78793
    United States

    Site Not Available

  • Novartis Investigative Site

    Austin, Texas 78793
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Bellaire, Texas 77401
    United States

    Active - Recruiting

  • Retina Consultants TX Rsrch Ctr

    Bellaire, Texas 77401
    United States

    Site Not Available

  • Novartis Investigative Site

    Dallas, Texas 75231
    United States

    Active - Recruiting

  • Retina Foundation

    Dallas, Texas 75231
    United States

    Site Not Available

  • Novartis Investigative Site

    Fort Worth, Texas 76104
    United States

    Active - Recruiting

  • Texas Retina Associates

    Fort Worth, Texas 76104
    United States

    Site Not Available

  • Novartis Investigative Site

    Houston, Texas 77030
    United States

    Active - Recruiting

  • Retina Consultants of Houston PA

    Houston, Texas 77030
    United States

    Site Not Available

  • Retina Consultants of Houston PA .

    Houston, Texas 77030
    United States

    Site Not Available

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