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

NCT ID: NCT05230537

Last Updated: 2025-07-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

170 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-02-17

Study Completion Date

2026-10-02

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Age-Related Macular Degeneration

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a multicenter, randomized, participant and investigator masked, placebo controlled, proof-of-concept study to assess the safety and efficacy of LNP023 in participants with early to intermediate age-related macular degeneration (e/iAMD) in one eye and neovascular age-related macular degeneration (nAMD) in the other eye.
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors
Investigator and Participant

Study Groups

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Iptacopan (LNP023)

Iptacopan (LNP023) oral use capsules

Group Type EXPERIMENTAL

Iptacopan (LNP023)

Intervention Type DRUG

oral capsules

Placebo

Placebo matched to study drug, oral use capsules

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

oral capsules

Interventions

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Iptacopan (LNP023)

oral capsules

Intervention Type DRUG

Placebo

oral capsules

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

50 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Retina Consultants of Orange County

Fullerton, California, United States

Site Status

Salehi Retina Institute

Huntington Beach, California, United States

Site Status

Martel Eye Medical Group

Rancho Cordova, California, United States

Site Status

Retinal Consultants Medical Group Inc

Sacramento, California, United States

Site Status

California Retina Consultants

Santa Barbara, California, United States

Site Status

Southwest Retina Research Center

Durango, Colorado, United States

Site Status

Advanced Research LLC

Deerfield Beach, Florida, United States

Site Status

Retina Center Of South Florida

Delray Beach, Florida, United States

Site Status

Retina Vitreous Associates of Florida

St. Petersburg, Florida, United States

Site Status

Midwest Eye Institute

Indianapolis, Indiana, United States

Site Status

Retina Associates New Orleans

New Orleans, Louisiana, United States

Site Status

Retina Care Center

Baltimore, Maryland, United States

Site Status

Opthamalic Consultants of Boston

Boston, Massachusetts, United States

Site Status

Envision Ocular LLC

Bloomfield, New Jersey, United States

Site Status

Duke Eye Center

Durham, North Carolina, United States

Site Status

Retina Northwest PC

Portland, Oregon, United States

Site Status

Charles Retina Institute

Germantown, Tennessee, United States

Site Status

Austin Research Center for Retina

Austin, Texas, United States

Site Status

Retina Consultants TX Rsrch Ctr

Bellaire, Texas, United States

Site Status

Retina Foundation

Dallas, Texas, United States

Site Status

Texas Retina Associates

Fort Worth, Texas, United States

Site Status

Retina Consultants of Houston PA

Houston, Texas, United States

Site Status

Novartis Investigative Site

Harbin, Heilongjiang, China

Site Status

Novartis Investigative Site

Tianjin, Tianjin Municipality, China

Site Status

Novartis Investigative Site

Shanghai, , China

Site Status

Emanuelli Research and Development Center LLC

Arecibo, , Puerto Rico

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Novartis Investigative Site

Southampton, , United Kingdom

Site Status

Countries

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United States China Puerto Rico United Kingdom

References

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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.

Reference Type DERIVED
PMID: 37314061 (View on PubMed)

Other Identifiers

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2021-001797-31

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CLNP023E12201

Identifier Type: -

Identifier Source: org_study_id

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