Iptacopan for Age-Related Macular Degeneration

Not currently recruiting at 60 trial locations

Overseen By Karl Csaky

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Novartis Pharmaceuticals

Disqualifiers: Cardiac arrhythmias, Stroke, Myocardial infarction, End stage kidney disease, Malignancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if Iptacopan, a new potential drug, can prevent early or intermediate age-related macular degeneration (AMD) from worsening. The goal is to stop these eyes from developing more serious issues that can lead to vision loss. Participants will receive either Iptacopan or a placebo, a pill without the active drug. This trial suits individuals aged 50 or older with early or intermediate AMD in one eye and a more severe form in the other. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to important findings.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that Iptacopan is likely to be safe for humans?

Research has shown that Iptacopan has undergone testing in other studies to assess its safety. One study focused on patients with paroxysmal nocturnal hemoglobinuria (PNH) and found positive results. Specifically, patients in that study tolerated Iptacopan well without major safety issues. This suggests it might also be safe for other conditions, such as age-related macular degeneration (AMD), although specific data for AMD is still being collected.

As this trial is in an early stage, the treatment is still under evaluation to ensure its safety and effectiveness for AMD. However, positive safety results from other uses of Iptacopan provide some reassurance. Prospective participants should consult a healthcare provider about any concerns before deciding to join the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for AMD?

Unlike the standard treatments for age-related macular degeneration, which often involve injections directly into the eye, Iptacopan (LNP023) is an oral capsule, making it potentially more comfortable and convenient for patients. Most current therapies aim to manage symptoms by inhibiting vascular endothelial growth factor (VEGF). However, Iptacopan works differently by targeting the complement system, a part of the immune system that's believed to contribute to AMD progression. Researchers are excited because this novel approach could provide an alternative pathway to slow or stop the disease without the need for frequent eye injections.

What evidence suggests that Iptacopan could be an effective treatment for age-related macular degeneration?

Research has shown that Iptacopan, which participants in this trial may receive, might help prevent the progression of age-related macular degeneration (AMD). This treatment blocks a part of the immune system that can harm the eye. In studies on other health issues, Iptacopan demonstrated good results, suggesting it could also be effective for AMD. Early evidence indicates it might stop or slow damage to the retina, the part of the eye responsible for vision. Although more research is needed specifically for AMD, these findings are promising for individuals in the early or middle stages of the disease. ^{2 3 4 6 7}

Who Is on the Research Team?

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

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

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

I have been vaccinated against Haemophilius influenzae if it was available and recommended.

Exclusion Criteria

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.

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

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Iptacopan or placebo oral capsules

24 months

Regular visits throughout the treatment period

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Iptacopan (LNP023)
• Placebo

Trial Overview 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.

How Is the Trial Designed?

Group I: Iptacopan (LNP023) Experimental Treatment 1 Intervention

Iptacopan (LNP023) oral use capsules

Group II: Placebo Placebo Group 1 Intervention

Placebo matched to study drug, oral use capsules

Iptacopan (LNP023) is already approved in United States for the following indications:

Approved in United States as Fabhalta for:

• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Primary Immunoglobulin A Nephropathy (IgAN)

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Published Research Related to This Trial

In a prospective, randomized trial involving 92 patients with wet age-related macular degeneration, the combination therapy of photodynamic therapy (PDT) and intravitreal triamcinolone (IVTA) did not show a significant improvement in visual acuity compared to PDT alone after 6 months.

All treatment groups experienced a decrease in retinal thickness, with significant results in the combination therapy groups, but there were no significant differences in re-treatment rates or cataract progression among the groups.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[ITAP - 6-month results of a prospective, randomised phase 3 study for evaluation of the combination therapy of PDT and intravitreal triamcinolone in exudative AMD]. Gamulescu, MA., Schubert, K., Thormann, S., et al.[2013]

In a study of 205 patients with exudative age-related macular degeneration, a single intravitreal injection of triamcinolone acetonide led to significant initial improvements in visual acuity, but these benefits diminished over time, with no apparent advantage at 12 months post-injection.

While intraocular pressure increased significantly in the first 6 months after the injection, it returned to baseline levels by 9 months, indicating a temporary side effect that resolved without long-term complications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Follow-up after intravitreal triamcinolone acetonide for exudative age-related macular degeneration. Jonas, JB., Spandau, UH., Kamppeter, BA., et al.[2013]

Intravitreal triamcinolone acetonide (iTAAC) injections combined with photodynamic therapy (PDT) for treating age-related macular degeneration (AMD) showed that 50% of patients maintained stable vision after one year, with 7% gaining significant vision improvement of 30 letters or more.

The treatment was relatively safe, with mild side effects reported, including intraocular pressure elevation in 28.5% of patients and cataract progression in 50% of those with natural lenses, indicating a need for monitoring.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravitreal triamcinolone with photodynamic therapy for subfoveal choroidal neovascularisation in age related macular degeneration. Rechtman, E., Danis, RP., Pratt, LM., et al.[2019]

In a study involving 30 patients with age-related macular degeneration, the combination of photodynamic therapy (PDT) and intravitreal triamcinolone acetonide (IVTA) effectively stabilized visual acuity over 12 months, while PDT alone resulted in a significant decline in visual acuity.

The combination therapy also reduced the need for retreatments, with a treatment rate of 1.13 in the PDT plus IVTA group compared to 3.6 in the PDT alone group, although there were some safety concerns, including cataract progression and elevated intraocular pressure in some patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Triamcinolone acetonide as adjunctive treatment to verteporfin in neovascular age-related macular degeneration: a prospective randomized trial. Chaudhary, V., Mao, A., Hooper, PL., et al.[2018]

Intravitreal triamcinolone acetonide (IVTA) injections are commonly used in the UK for treating macular oedema, with 33% of surveyed ophthalmologists reporting its use in 2006, totaling 3899 injections.

The study found that at least 1.1% of IVTA injections resulted in severe intraocular pressure (IOP) rises that required laser treatment or surgery, highlighting a significant safety concern associated with this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

UK national survey of current practice and experience of intravitreal triamcinolone acetonide. Jain, S., Hurst, JR., Thompson, JR., et al.[2016]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05230537

A Masked, Placebo-controlled Study to Assess Iptacopan ... 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.

2. synapse.patsnap.com synapse.patsnap.com/article/how-does-iptacopancompare-with-other-treatments-for-amd

How does Iptacopancompare with other treatments for AMD? The resulting treatment landscape is highly dynamic, with an emphasis on not only halting disease progression but also improving cost- ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10266126/

Complement inhibitors for age‐related macular degeneration Outcome measures evaluated at one year included change in best‐corrected visual acuity (BCVA), untransformed and square root‐transformed geographic atrophy (GA) ...

4. retinalphysician.com retinalphysician.com/issues/2022/may/clinical-trial-update/

Clinical Trial Update May 2022 Purpose: The purpose of this study is to assess the effect of Iptacopan to prevent conversion of early or intermediate age-related macular ...

5. clinicaltrialsarena.com clinicaltrialsarena.com/news/novartis-iptacopan-pnh-2/

Novartis reports results from Phase IIIB study of iptacopan ... Novartis has reported positive outcomes from the multi-centre Phase IIIB APPULSE-PNH study of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05230537

A Masked, Placebo-controlled Study to Assess Iptacopan ... 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 ...

7. withpower.com withpower.com/trial/phase-2-age---related-macular-degeneration-amd-1-2022-c4bd0

Iptacopan for Age-Related Macular Degeneration This trial tests if Iptacopan capsules can prevent worsening of early or intermediate AMD in people who already have severe AMD in one eye.