Age-Related Macular DegenerationPhase 2NCT05230537
A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration
A Phase 2 study testing Iptacopan (LNP023) in people with Age-Related Macular Degeneration, run by Novartis Pharmaceuticals at 28 sites in 4 countries.
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. Read more about this study
- Who is it for?
- Ages 50 to 100 years
- Conditions the study is for
- Age-Related Macular Degeneration
- What is being tested?
- Iptacopan (LNP023)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 168 participants
- Where
- 28 sitesin 4 countries
- Study dates
- 17 Feb 2022to 30 Sep 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 12Show fewer
- 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.
Trial locations and contacts
28 sites in 4 countries.
28 sites in 4 countries
United States22 sites
Austin Research Center for Retina
Austin, Texas, 78793
Retina Care Center
Baltimore, Maryland, 21209
Retina Consultants TX Rsrch Ctr
Bellaire, Texas, 77401
Envision Ocular LLC
Bloomfield, New Jersey, 07003
Opthamalic Consultants of Boston
Boston, Massachusetts, 02114
Midwest Eye Institute
Carmel, Indiana, 46032
Retina Foundation
Dallas, Texas, 75231
Advanced Research LLC
Deerfield Beach, Florida, 33064
Retina Center Of South Florida
Delray Beach, Florida, 33484
Southwest Retina Research Center
Durango, Colorado, 81303
Duke Eye Center
Durham, North Carolina, 27705
Texas Retina Associates
Fort Worth, Texas, 76104
Retina Consultants of Orange County
Fullerton, California, 92835
Charles Retina Institute
Germantown, Tennessee, 38138
Retina Consultants of Houston PA
Houston, Texas, 77030
Salehi Retina Institute
Huntington Beach, California, 92647
Retina Associates New Orleans
Metairie, Louisiana, 70001
Retina Northwest PC
Portland, Oregon, 97210
Martel Eye Medical Group
Rancho Cordova, California, 95670
Retinal Consultants Medical Group Inc
Sacramento, California, 95841
California Retina Consultants
Santa Barbara, California, 93103
Retina Vitreous Associates of Florida
St. Petersburg, Florida, 33711
China3 sites
Novartis Investigative Site
Harbin, Heilongjiang, 150001
Novartis Investigative Site
Shanghai, 200080
Novartis Investigative Site
Tianjin, Tianjin Municipality, 300020
United Kingdom2 sites
Novartis Investigative Site
London, SE5 9RS
Novartis Investigative Site
Southampton, SO16 6YD
Puerto Rico1 site
Emanuelli Research and Development Center LLC
Arecibo, 00612
About this study
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
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.
Read the 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.
- Conditions
- Age-Related Macular Degeneration
- Being tested
- Iptacopan (LNP023), Placebo
- 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
- Medical subject headings
- Macular Degeneration, Vision Disorders, Geographic Atrophy, Wet Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Iptacopan (LNP023)Drug
oral capsules
PlaceboDrug
oral capsules
| Study group | Role | What they receive |
|---|---|---|
| Iptacopan (LNP023) | Experimental | Iptacopan (LNP023) oral use capsules Receives: Drug: Iptacopan (LNP023) |
| Placebo | Placebo comparator | Placebo matched to study drug, oral use capsules Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline/Day 1 through Month 24
Secondary outcomes (7)
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.
Measured: Baseline/Day 1 through Month 24
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.
Measured: Baseline/Day 1 through Month 24
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.
Measured: Baseline/Day 1 through Month 24
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.
Measured: Baseline/Day 1 through Month 24
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.
Measured: Baseline/Day 1 through Month 24
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.
Measured: Baseline/Day 1 through Month 24
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.
Measured: Baseline/Day 1 through Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2022
- Study start17 Feb 2022
- Primary completion19 Aug 2026
- Study completion30 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Age-Related Macular DegenerationStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 168 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05230537
- Sponsor's protocol ID
- CLNP023E12201
- Status last verified
- Oct 2026
- First submitted
- 28 Jan 2022
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Publications
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.
PubMed 37314061
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05230537
- Acronym / sponsor ID
- CLNP023E12201
- Conditions
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 168 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 17 Feb 2022 (actual)
- Primary completion
- 19 Aug 2026 (actual)
- Study completion
- 30 Sep 2026 (actual)
- First posted
- 9 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Iptacopan (LNP023) | Experimental | Drug: Iptacopan (LNP023)Iptacopan (LNP023) oral use capsules |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo matched to study drug, oral use capsules |
Who may qualify
- Ages
- 50 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Iptacopan (LNP023)oral capsules | Drug | Iptacopan (LNP023) |
| Placebooral capsules | Drug | Placebo |
Primary outcome
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
Time frame: Baseline/Day 1 through Month 24
OCT and other imaging will be performed using spectral domain OCT or swept source OCT machines
7 secondary outcomes
The incidence of ocular and non-ocular adverse events (AEs)
Time frame: Baseline/Day 1 through Month 24
Change in Early Treatment Diabetic Retinopathy Study (ETDRS) (Standard Luminance) best corrected visual acuity (BCVA) scores in the early/intermediate AMD eye
Time frame: Baseline/Day 1 through Month 24
Change in ETDRS low luminance visual acuity (LLVA) scores in the early/intermediate AMD eye
Time frame: Baseline/Day 1 through Month 24
Change in contrast sensitivity (CS) scores in the early/intermediate AMD eye
Time frame: Baseline/Day 1 through Month 24
Change in Standard luminance manifold contrast vision meter contrast sensitivity (MCVM-CS) scores in the early/intermediate AMD eye
Time frame: Baseline/Day 1 through Month 24
Pharmacokinetics - concentrations of LNP023 related to trough samples
Time frame: Baseline/Day 1 through Month 24
Change in low luminance manifold contrast vision meter (MCVM) contrast sensitivity scores in the early/intermediate AMD eye
Time frame: Baseline/Day 1 through Month 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Derived from this study: PubMed 37314061Tzoumas 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.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
28 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Iptacopan (LNP023) in people with Age-Related Macular Degeneration, run by Novartis Pharmaceuticals at 28 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, China, United Kingdom, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: Baseline/Day 1 through Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05230537, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.