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

Primary Purpose

Age-Related Macular Degeneration

Status

Recruiting

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Iptacopan (LNP023)

Placebo

About this trial

This is an interventional treatment trial for Age-Related Macular Degeneration focused on measuring Age-related macular degeneration, Macular degeneration, vision loss, macula damage, retina damage, dry macular degeneration, wet macular degeneration, AMD, Best Corrected Visual Acuity, Neovascular AMD, OCT

Eligibility Criteria

50 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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:

Concomitant medical or ocular conditions which could compromise visual acuity, require planned medical or surgical intervention during the study period, preclude scheduled study visits, completion of the study, or safe administration of the investigational product, including intraocular surgery, cataract and vitreoretinal surgery in the study eye within 3 months prior to Baseline/Day 1 and the presence of significant media opacity, eye movement disorder (nystagmus), severe ptosis, extraocular motility restriction or head tremor.
History of clinically significant electrocardiogram (ECG) abnormalities, or any of the following ECG abnormalities at screening or Baseline/Day 1 visit:
- QT interval corrected by Fridericia's formula (QTcF) >450 msec (males)
- QTcF >460 msec (females)
- 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 kidney failure including end stage renal 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
Chronic infection with Hepatitis B or Hepatitis C.
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.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Iptacopan (LNP023)

Placebo

Arm Description

Iptacopan (LNP023) oral use capsules

Placebo matched to study drug, oral use capsules

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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.

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.

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.

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.

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.

Pharmacokinetics - concentrations of LNP023 related to trough samples

Concentrations of LNP023 related to trough samples

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.

Full Information

NCT ID

NCT05230537

First Posted

January 28, 2022

Last Updated

July 18, 2023

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT05230537

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2023

Overall Recruitment Status

Recruiting

Study Start Date

February 17, 2022 (Actual)

Primary Completion Date

September 2, 2026 (Anticipated)

Study Completion Date

September 3, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Age-Related Macular Degeneration

Keywords

Age-related macular degeneration, Macular degeneration, vision loss, macula damage, retina damage, dry macular degeneration, wet macular degeneration, AMD, Best Corrected Visual Acuity, Neovascular AMD, OCT

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

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.

Masking

ParticipantInvestigatorOutcomes Assessor

Masking Description

Investigator and Participant

Allocation

Randomized

Enrollment

146 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Iptacopan (LNP023)

Arm Type

Experimental

Arm Description

Iptacopan (LNP023) oral use capsules

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Placebo matched to study drug, oral use capsules

Intervention Type

Drug

Intervention Name(s)

Iptacopan (LNP023)

Intervention Description

oral capsules

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

oral capsules

Primary Outcome Measure Information:

Title

Description

OCT and other imaging will be performed using spectral domain OCT or swept source OCT machines

Time Frame