Last Update: May 17, 2024
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
ClinicalTrials.gov Identifier:
Novartis Reference Number:CLNP023E12201
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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. 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.

Age-Related Macular Degeneration
Phase2
Recruiting
146
Feb 17, 2022
Oct 12, 2026
All
50 Years - (Adult, Older Adult)

Interventions

Drug

Iptacopan (LNP023)

oral capsules
Drug

Placebo

oral capsules

Eligibility 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.

Study Location

Novartis Investigative Site

Recruiting

Harbin City,Heilongjiang,150000,China

Novartis Investigative Site

Recruiting

Shanghai,200080,China

Novartis Investigative Site

Recruiting

Tianjin,Tianjin,300020,China

Novartis Investigative Site

Recruiting

Gloucester,Gl1 3nn,United Kingdom

Novartis Investigative Site

Recruiting

London,Se5 9rs,United Kingdom

Novartis Investigative Site

Recruiting

Southampton,So16 6yd,United Kingdom

Advanced Research LLC Research

Recruiting

Deerfield Beach,Florida,33064,United States

Retina Associates New Orleans .

Recruiting

New Orleans,Louisiana,70115-8139,United States

Retina Northwest PC .

Recruiting

Portland,Oregon,97210,United States

Austin Research Center for Retina .

Recruiting

Austin,Texas,78793,United States

Retina Care Center Research

Recruiting

Baltimore,Maryland,21209,United States

Retinal Consultants Medical Group, Inc

Recruiting

Sacramento,California,95841,United States

California Retina Consultants .

Recruiting

Santa Barbara,California,93103,United States

Retina Center Of South Florida

Recruiting

Delray Beach,Florida,33484,United States

Southwest Retina Research Center

Recruiting

Durango,Colorado,81303,United States

Charles Retina Institute

Recruiting

Germantown,Tennessee,38138,United States

Retina Consultants of Houston PA .

Recruiting

Houston,Texas,77030,United States

Texas Retina Associates

Recruiting

Fort Worth,Texas,76104,United States

Duke Eye Center Research

Recruiting

Durham,North Carolina,27705,United States

Retina Consultants of Orange County

Recruiting

Fullerton,California,92835,United States

Vitreous Retina Macula Consultants Research

Recruiting

New York,New York,10022,United States

Envision Ocular LLC

Recruiting

Bloomfield,New Jersey,07003,United States

Retina Consultants TX Rsrch Ctr

Recruiting

Bellaire,Texas,77401,United States

Martel Eye Medical Group

Recruiting

Rancho Cordova,California,95670,United States

Retina Foundation

Recruiting

Dallas,Texas,75231,United States

Worldwide Contacts

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Novartis Pharmaceuticals

Novartis Pharmaceuticals