Iptacopan for Age-Related Macular Degeneration

Recruiting at 49 trial locations

Overseen By Karl Csaky

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: Novartis Pharmaceuticals

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

Prior Safety Data

Breakthrough Therapy

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

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.

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.

How is the drug Iptacopan different from other treatments for age-related macular degeneration?

Iptacopan is unique because it targets the complement system, a part of the immune system, which is not the focus of traditional treatments like photodynamic therapy or intravitreal triamcinolone acetonide injections. This novel approach may offer a new way to manage age-related macular degeneration by addressing underlying immune processes. ^{1 2 3 4 5}

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

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.

Trial Timeline

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

Treatment Details

Interventions

• Iptacopan (LNP023) (Complement Inhibitor)
• Placebo (Drug)

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.

Participant Groups

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

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

Findings from Research

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 (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]

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 (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]

References

1. Germany 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]. [2013]

2. England pubmed.ncbi.nlm.nih.gov

Follow-up after intravitreal triamcinolone acetonide for exudative age-related macular degeneration. [2013]

3. England pubmed.ncbi.nlm.nih.gov

UK national survey of current practice and experience of intravitreal triamcinolone acetonide. [2016]

4. United States pubmed.ncbi.nlm.nih.gov

Triamcinolone acetonide as adjunctive treatment to verteporfin in neovascular age-related macular degeneration: a prospective randomized trial. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Intravitreal triamcinolone with photodynamic therapy for subfoveal choroidal neovascularisation in age related macular degeneration. [2019]