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 laser therapy. Anti-VEGF treatments, such as ranibizumab and aflibercept, can cause side effects like eye pain, increased intraocular pressure, and inflammation. Photodynamic therapy may lead to temporary vision changes and photosensitivity. Laser therapy can result in scarring and potential vision loss. Complement Factor D Inhibitors, like Danicopan, are being studied for their efficacy and safety, with potential side effects including injection site reactions and mild ocular discomfort.

What prior FDA approvals exist?

The FDA has approved several treatments for Age-Related Macular Degeneration (AMD), primarily focusing on anti-VEGF (vascular endothelial growth factor) therapies. These include drugs like Ranibizumab (Lucentis), Aflibercept (Eylea), and Bevacizumab (Avastin), which inhibit the growth of abnormal blood vessels in the eye. While Danicopan is a Complement Factor D Inhibitor, targeting a different pathway in the complement system, it represents a novel approach compared to the existing anti-VEGF therapies. Other complement pathway inhibitors, such as Pegcetacoplan (Empaveli), have also been explored for AMD, though their primary indications may differ.

What other types of research exist?

A promising alternative to Danicopan for Age-Related Macular Degeneration patients is aflibercept, which has demonstrated efficacy in managing wet AMD. Other potential treatments include ranibizumab and ongoing research into gene therapies and other pharmacological agents targeting different pathways involved in AMD.

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Complement Inhibitor

Danicopan for Age-Related Macular Degeneration

Phase 2

Waitlist Available

Research Sponsored by Alexion Pharmaceuticals, Inc.

Eligibility Criteria Checklist

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

Must have

Vaccination for Neisseria meningitidis

Presentation of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) in at least 1 eye

Must not have

Previous receipt of any stem cell/gene therapy for any ophthalmological condition in either eye

Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan), or any of its excipients

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial is testing a medication called danicopan to see if it can help people with a specific type of eye disease called Geographic Atrophy, which is related to Age-related Macular Degeneration. The study will check how well the medication works, how safe it is, and how the body handles it.

Who is the study for?

This trial is for individuals with a condition called Geographic Atrophy (GA) that's due to Age-Related Macular Degeneration (AMD). Participants must have received a specific vaccination, be able to consent, and have GA lesions outside the central part of their retina. People are excluded if they've had other treatments or studies for GA in the last 3 months, have complement deficiencies, certain medical conditions deemed risky by the investigator, previous stem cell/gene therapy for eye conditions, active ocular diseases affecting vision assessment or allergies to study drug components.

What is being tested?

The study tests different doses of Danicopan (100 mg twice daily, 200 mg twice daily, and 400 mg once daily) against a placebo to see which is more effective and safe over two years. It aims to find out how well Danicopan works on GA secondary to AMD and monitors participants' reactions over time after taking it.

What are the potential side effects?

While specific side effects aren't listed here, common ones may include allergic reactions like rashes or itching due to hypersensitivity; potential risks could also involve issues related to immune response given Danicopan targets complement pathways involved in inflammation.

Eligibility Criteria

Inclusion Criteria

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

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I have been vaccinated against meningitis.

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I have vision loss in one eye due to AMD.

Exclusion Criteria

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

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I have received stem cell or gene therapy for an eye condition.

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I am not allergic to the trial drug or its ingredients.

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My vision loss is not due to AMD.

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I do not have eye conditions that could affect my vision or interfere with the study.

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I have a complement system deficiency.

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I have had eye injections for blood vessel issues in my study eye.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2024 Phase 3 trial • 88 Patients • NCT04469465

24%

Pyrexia

20%

COVID-19

15%

Headache

13%

Nasopharyngitis

Asthenia

Urinary tract infection

Fatigue

Pain in extremity

Breakthrough haemolysis

Cough

Non-cardiac chest pain

Nausea

Productive cough

Dizziness

Back pain

Contusion

Haemolysis

Anaemia

Thrombocytopenia

Hypertension

Abdominal pain

Vomiting

Stent-graft endoleak

Haemoglobin decreased

Platelet count decreased

Pulmonary embolism

Insomnia

Chromaturia

Dyspnoea

Diarrhoea

Arthralgia

Tachycardia

Pulmonary haemorrhage

Invasive ductal breast carcinoma

100%

80%

60%

40%

20%

Study treatment Arm

Placebo-Danicopan (TP2)

Danicopan-Danicopan (LTE)

Danicopan-Danicopan (TP2)

Placebo-Danicopan (LTE)

Danicopan (TP1)

Placebo (TP1)

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Danicopan: 400 mgExperimental Treatment1 Intervention

Participants will receive danicopan 400 mg qd during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group II: Danicopan: 200 mgExperimental Treatment1 Intervention

Participants will receive danicopan 200 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group III: Danicopan: 100 mgExperimental Treatment1 Intervention

Participants will receive danicopan 100 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.

Group IV: PlaceboPlacebo Group2 Interventions

Participants will receive matching placebo and will be re-randomized to one of the active treatment groups at Week 52, or to the optimal dose, if already identified.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Danicopan

2020

Completed Phase 3

~680

0 / 8Eligibility 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

Common treatments for Age-Related Macular Degeneration (AMD) include complement inhibitors like Danicopan, which target the complement system to reduce inflammation and tissue damage associated with geographic atrophy. Anti-VEGF therapies are also widely used to inhibit abnormal blood vessel growth and leakage in the retina. These treatments are essential for slowing disease progression and preserving vision, thereby improving the quality of life for AMD patients.