What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD) include anti-VEGF (vascular endothelial growth factor) injections, photodynamic therapy, and complement inhibitors like Iptacopan. Anti-VEGF treatments, such as ranibizumab and aflibercept, can cause side effects like eye pain, increased intraocular pressure, and rare cases of retinal detachment or endophthalmitis. Photodynamic therapy may lead to temporary vision changes and photosensitivity. Complement inhibitors, which target the complement system involved in inflammation, can have side effects including eye irritation, conjunctival hemorrhage, and potential systemic effects like hypertension. It is important to discuss these potential side effects with a healthcare provider to determine the best treatment plan.

What prior FDA approvals exist?

FDA-approved treatments for Age-Related Macular Degeneration (AMD) primarily include anti-VEGF (vascular endothelial growth factor) therapies such as ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin). These drugs work by inhibiting the growth of abnormal blood vessels in the retina. While Iptacopan, a Complement Factor B Inhibitor, is being studied for its potential to prevent the progression of AMD, it represents a different mechanism of action compared to the currently approved anti-VEGF therapies.

What other types of research exist?

Promising alternatives to Iptacopan for AMD patients include gene therapies like voretigene neparvovec (Luxturna) for specific genetic mutations, and other complement inhibitors such as pegcetacoplan. Additionally, anti-VEGF therapies like brolucizumab and faricimab, which target vascular endothelial growth factors, are also being explored for their efficacy in treating AMD.

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Complement Inhibitor

Iptacopan for Age-Related Macular Degeneration

Phase 2

Recruiting

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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 early or intermediate age-related macular degeneration (AMD) in the study eye as determined by the investigator on fundus examination

Must not have

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline/day 1 through month 24

Summary

This trial tests if Iptacopan capsules can prevent worsening of early or intermediate AMD in people who already have severe AMD in one eye. The goal is to see if the medication can protect eye cells and slow down vision loss.

Who is the study for?

This trial is for men and women over 50 with early or intermediate age-related macular degeneration (AMD) in one eye, who are at high risk as shown by OCT imaging. Participants must be vaccinated against certain infections before treatment starts. Those with recent eye surgery, significant heart issues, kidney failure, cancer history, immune deficiencies or known allergies to study drugs cannot join.

What is being tested?

The trial is testing Iptacopan's effectiveness in preventing the progression from early or intermediate AMD to more advanced stages of the disease. Participants will either receive Iptacopan or a placebo without knowing which one they're getting to compare outcomes fairly.

What are the potential side effects?

While specific side effects of Iptacopan aren't listed here, common ones may include reactions at the injection site, potential infection risks due to immunosuppression if applicable, and possibly others based on similar medications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My eye condition is at risk according to a special eye scan.

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I have been diagnosed with early or intermediate AMD in one eye.

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My other eye has been diagnosed with wet age-related macular degeneration.

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I have been vaccinated against meningitis and pneumonia before starting LNP023 treatment.

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I am 50 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I haven't had a stroke or heart attack in the last 6 months and don't have serious heart rhythm problems or severe lung blood pressure issues.

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I have had a kidney transplant or need dialysis for end-stage kidney disease.

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My family has a history of long QT syndrome or Torsades de Pointes.

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I do not have an active Hepatitis B or C infection.

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I have a history of immune system diseases or tested positive for HIV.

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I currently have active tuberculosis.

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I have had cancer in any part of my body before.

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I have had a solid organ or bone marrow transplant.

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I have had meningitis more than once or got meningococcal infection despite being vaccinated.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline/day 1 through month 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline/day 1 through month 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline/day 1 through month 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

Change in ETDRS low luminance visual acuity (LLVA) scores in the early/intermediate AMD eye

Change in Early Treatment Diabetic Retinopathy Study (ETDRS) (Standard Luminance) best corrected visual acuity (BCVA) scores in the early/intermediate AMD eye

Change in Standard luminance manifold contrast vision meter contrast sensitivity (MCVM-CS) scores in the early/intermediate AMD eye

+4 more

Side effects data

From 2023 Phase 3 trial • 40 Patients • NCT04820530

30%

Headache

18%

COVID-19

18%

Upper respiratory tract infection

15%

Diarrhoea

Pyrexia

Abdominal pain

Iron deficiency

Constipation

Vomiting

Breakthrough haemolysis

Cataract

Type 2 diabetes mellitus

Malignant melanoma

Infection

Pneumonia

Pneumonia bacterial

100%

80%

60%

40%

20%

Study treatment Arm

LNP023 200mg b.i.d.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Iptacopan (LNP023)Experimental Treatment1 Intervention

Iptacopan (LNP023) oral use capsules

Group II: PlaceboPlacebo Group1 Intervention

Placebo matched to study drug, oral use capsules

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Iptacopan (LNP023)

2021

Completed Phase 3

~40

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Age-Related Macular Degeneration (AMD) treatments often target the underlying mechanisms that contribute to retinal damage. Complement inhibitors, such as Iptacopan, work by inhibiting components of the complement system, specifically Complement Factor B, which plays a role in inflammation and tissue damage in AMD. By reducing this inflammation, these inhibitors can potentially slow the progression of AMD. Other common treatments include anti-VEGF (vascular endothelial growth factor) therapies, which reduce abnormal blood vessel growth and leakage in the retina, thereby preserving vision. These treatments are crucial for AMD patients as they address the root causes of retinal damage, aiming to prevent vision loss and improve quality of life.