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

Age-Related Macular Degeneration Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05230537
• Other study ID #: CLNP023E12201
• Secondary ID: 2021-001797-31
• Status: Recruiting
• Phase: Phase 2
• Start date: February 17, 2022
• Est. completion date: October 12, 2026

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: 1-888-669-6682
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 146
• Est. completion date: October 12, 2026
• Est. primary completion date: October 12, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Age-Related Macular Degeneration
• Macular Degeneration

Intervention

Drug:
• Iptacopan (LNP023)
oral capsules
• Placebo
oral capsules

Locations

Country	Name	City	State
China	Novartis Investigative Site	Harbin City	Heilongjiang
China	Novartis Investigative Site	Shanghai
China	Novartis Investigative Site	Tianjin	Tianjin
Puerto Rico	Emanuelli Research and Development Center LLC	Arecibo
United Kingdom	Novartis Investigative Site	Gloucester
United Kingdom	Novartis Investigative Site	London
United Kingdom	Novartis Investigative Site	Southampton
United States	Austin Research Center for Retina .	Austin	Texas
United States	Retina Care Center Research	Baltimore	Maryland
United States	Retina Consultants TX Rsrch Ctr	Bellaire	Texas
United States	Envision Ocular LLC	Bloomfield	New Jersey
United States	Opthamalic Consultants of Boston	Boston	Massachusetts
United States	Retina Foundation	Dallas	Texas
United States	Advanced Research LLC	Deerfield Beach	Florida
United States	Advanced Research LLC Research	Deerfield Beach	Florida
United States	Retina Center Of South Florida	Delray Beach	Florida
United States	Southwest Retina Research Center	Durango	Colorado
United States	Duke Eye Center Research	Durham	North Carolina
United States	Texas Retina Associates	Fort Worth	Texas
United States	Retina Consultants of Orange County	Fullerton	California
United States	Charles Retina Institute	Germantown	Tennessee
United States	Retina Consultants of Houston PA .	Houston	Texas
United States	Salehi Retina Institute	Huntington Beach	California
United States	Midwest Eye Institute Research	Indianapolis	Indiana
United States	Novartis Investigative Site	Kingston	Pennsylvania
United States	Retina Associates New Orleans .	New Orleans	Louisiana
United States	Vitreous Retina Macula Consultants Research	New York	New York
United States	Retina Northwest PC .	Portland	Oregon
United States	Martel Eye Medical Group	Rancho Cordova	California
United States	Retinal Consultants Medical Group, Inc	Sacramento	California
United States	Retina Vitreous Associates of Florida	Saint Petersburg	Florida
United States	California Retina Consultants .	Santa Barbara	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  China,  Puerto Rico,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	The incidence of ocular and non-ocular adverse events (AEs)	An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.	Baseline/Day 1 through Month 24
Secondary	Change in Early Treatment Diabetic Retinopathy Study (ETDRS) (Standard Luminance) best corrected visual acuity (BCVA) scores in the early/intermediate AMD eye	Best corrected visual acuity (BCVA) will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart. The number of letters read correctly, for each eye, will be recorded. Participants at sites in some countries may conduct BCVA testing using numerical charts rather than letter charts.	Baseline/Day 1 through Month 24
Secondary	Change in ETDRS low luminance visual acuity (LLVA) scores in the early/intermediate AMD eye	ETDRS low luminance visual acuity (LLVA) scores will be measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart. The number of letters read correctly, for each eye, will be recorded. Participants at sites in some countries may conduct BCVA testing using numerical charts rather than letter charts.	Baseline/Day 1 through Month 24
Secondary	Change in contrast sensitivity (CS) scores in the early/intermediate AMD eye	Pelli-Robson contrast sensitivity measurements will be performed using a Pelli-Robson contrast sensitivity wall chart and recording the number of correct letters read.	Baseline/Day 1 through Month 24
Secondary	Change in Standard luminance manifold contrast vision meter contrast sensitivity (MCVM-CS) scores in the early/intermediate AMD eye	MCVM contrast sensitivity measurements will be performed using an automated device that uses an adaptive algorithm to rapidly measure a contrast sensitivity function in 5-10 minutes.	Baseline/Day 1 through Month 24
Secondary	Pharmacokinetics - concentrations of LNP023 related to trough samples	Bioanalytical determination of plasma LNP023 concentrations related to trough samples using a LC-MS (mass spectrometry) method.	Baseline/Day 1 through Month 24
Secondary	Change in low luminance manifold contrast vision meter (MCVM) contrast sensitivity scores in the early/intermediate AMD eye	MCVM contrast sensitivity measurements will be performed using an automated device that uses an adaptive algorithm to rapidly measure a contrast sensitivity function in 5-10 minutes.	Baseline/Day 1 through Month 24