What is the mechanism of action of this treatment?

The most common treatments for Stargardt Disease, such as Avacincaptad Pegol, focus on inhibiting Complement Factor C5. This mechanism works by blocking the activation of the complement system, which is part of the immune response that can contribute to inflammation and cell damage in the retina. By inhibiting Complement Factor C5, these treatments aim to reduce retinal inflammation and slow the progression of retinal degeneration. This is particularly important for Stargardt Disease patients as it helps preserve vision and delay the onset of severe visual impairment.

What side effects are associated with this type of intervention?

The most common treatments for Stargardt Disease, particularly those involving complement inhibition like avacincaptad pegol, generally focus on reducing the progression of retinal degeneration. Known side effects of such treatments can include injection-site reactions such as pain, erythema, and bruising. Additionally, there may be risks of ocular side effects like inflammation or infection. While specific data on avacincaptad pegol is limited, similar treatments have shown these types of adverse effects in clinical trials.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Stargardt Disease. Avacincaptad Pegol, which is being studied for its efficacy in inhibiting Complement Factor C5, represents a novel approach in this area. Current research is focused on exploring various potential treatments, including gene therapy and other complement inhibitors, but these are still in clinical trial phases.

What other types of research exist?

Promising novel alternatives to Avacincaptad Pegol for Stargardt Disease patients include gene therapy approaches such as AAV-mediated gene transfer, which aims to correct the underlying genetic defect, and stem cell-based therapies that focus on regenerating retinal cells. Additionally, other pharmacological agents targeting different pathways involved in retinal degeneration, such as neuroprotective agents and anti-inflammatory drugs, are also being explored.

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Complement Factor C5 Inhibitor

Zimura for Stargardt Disease

Phase 2

Waitlist Available

Research Sponsored by Astellas Pharma Global Development, Inc.

Eligibility Criteria Checklist

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

Must have

At least two pathogenic mutations of ABCA4 gene confirmed by a CLIA-certified laboratory

Be between 18 and 65 years old

Must not have

Macular atrophy secondary to any condition other than STGD1 in either eye

Any major surgical procedure within one month of trial entry or anticipated during the trial

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 18 months

Summary

This trial is testing an eye injection medication to help people with Stargardt disease, a genetic condition that leads to vision loss. The medication aims to block harmful processes in the eye to prevent further vision loss.

Who is the study for?

This trial is for individuals with Stargardt Disease who have a visual acuity between 20/20 and 20/200, and two confirmed mutations of the ABCA4 gene. It's not for those who've had recent eye surgery, diabetes, stroke within the last year, or major surgery planned during the trial. Pregnant or nursing women and people with certain allergies or other eye conditions are also excluded.

What is being tested?

The study is testing Zimura™ (a complement factor C5 inhibitor) against a sham treatment to see if it's safe and effective in treating autosomal recessive Stargardt disease (STGD1). Participants will be randomly assigned to receive either Zimura or a placebo-like procedure without active medication.

What are the potential side effects?

While specific side effects of Zimura aren't listed here, similar treatments may cause injection-related reactions like redness or discomfort at the site of injection, potential allergic reactions, increased risk of infection due to immune system suppression, and possibly inflammation inside the eye.

Eligibility Criteria

Inclusion Criteria

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

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I have two harmful mutations in the ABCA4 gene.

Exclusion Criteria

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

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I have macular atrophy not caused by STGD1 in either eye.

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I haven't had major surgery in the last month and don't expect any during the trial.

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I have eye conditions like inflammation or a macular hole.

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I am not pregnant or nursing.

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I have not had any treatments for STGD1, including gene or stem cell therapy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 18 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 18 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 18 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Mean rate of change in the area of ellipsoid zone defect

Secondary study objectives

Emergence of at least one new atrophic lesion (DDAF)

Mean change in best corrected visual acuity

Mean change in mesopic macular sensitivity

+10 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: avacincaptad pegolExperimental Treatment1 Intervention

Participants will receive avacincaptad pegol monthly for up to 17 Months.

Group II: ShamPlacebo Group1 Intervention

Participants will receive a matching sham monthly for up to 17 Months.

0 / 6Eligibility 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

The most common treatments for Stargardt Disease, such as Avacincaptad Pegol, focus on inhibiting Complement Factor C5. This mechanism works by blocking the activation of the complement system, which is part of the immune response that can contribute to inflammation and cell damage in the retina. By inhibiting Complement Factor C5, these treatments aim to reduce retinal inflammation and slow the progression of retinal degeneration. This is particularly important for Stargardt Disease patients as it helps preserve vision and delay the onset of severe visual impairment.

EFFICACY OF INTRAVITREAL INJECTION OF CONBERCEPT IN POLYPOIDAL CHOROIDAL VASCULOPATHY: Subgroup Analysis of the Aurora Study.Effect of lesion size, visual acuity, and lesion composition on visual acuity change with and without verteporfin therapy for choroidal neovascularization secondary to age-related macular degeneration: TAP and VIP report no. 1.