Zimura for Stargardt Disease

No longer recruiting at 52 trial locations

Overseen By Sonia Cruz

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Astellas Pharma Global Development, Inc.

Must not be taking: Vitamin A derivatives

Disqualifiers: Diabetes, Stroke, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

Duration: 17 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Zimura to determine its safety and effectiveness for people with Stargardt disease, a genetic eye condition that causes vision loss. It compares Zimura injections to a placebo (sham treatment) over 17 months. Suitable participants have Stargardt disease confirmed by specific genetic testing and experience some vision loss but not complete blindness. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you are participating in another study or have had certain treatments recently, you may not be eligible.

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

Research has shown that avacincaptad pegol, also known as Zimura, appears safe. Early data suggest that most patients tolerate this treatment well. One important study found fewer new cases of a type of eye disease called nAMD in patients treated with avacincaptad pegol, indicating the treatment likely doesn't cause major eye problems. Other studies have reported similar results, supporting the safety of avacincaptad pegol. While side effects can occur with any treatment, the current data are encouraging for those considering joining a trial. ^{1 2 3 4 5}

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

Unlike the standard treatments for Stargardt disease, which primarily focus on managing symptoms, Zimura (avacincaptad pegol) targets the underlying causes of the condition. Zimura is unique because it inhibits complement factor C5, a component involved in the inflammatory processes that can lead to retinal damage. This mechanism of action is different from current options, offering the potential to slow or halt disease progression rather than just addressing its symptoms. Researchers are excited about Zimura because it represents a promising new approach that could significantly improve outcomes for patients with Stargardt disease.

What evidence suggests that Zimura might be an effective treatment for Stargardt disease?

Research has shown that avacincaptad pegol, also known as Zimura, may slow certain eye conditions. Specifically, it has slowed the progression of geographic atrophy in age-related macular degeneration (AMD). This trial will evaluate avacincaptad pegol for Stargardt disease, which resembles AMD. The treatment blocks certain proteins in the immune system that can harm the eyes. While direct evidence for Stargardt disease remains limited, the positive results in AMD are encouraging. ^{1 2 4 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Astellas Pharma Global Development, Inc.

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have two harmful mutations in the ABCA4 gene.

Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Exclusion Criteria

I have not had a stroke in the last year.

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

I have eye conditions like inflammation or a macular hole.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive avacincaptad pegol or sham intravitreal injection monthly

17 months

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 month

What Are the Treatments Tested in This Trial?

Interventions

• Zimura

Trial Overview 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.

How Is the Trial Designed?

Group I: avacincaptad pegol Experimental Treatment 1 Intervention

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

Group II: Sham Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Astellas Pharma Global Development, Inc.

Lead Sponsor

Trials

204

Recruited

123,000+

Tadaaki Taniguchi

Astellas Pharma Global Development, Inc.

Chief Medical Officer

M.D., Ph.D.

Naoki Okamura

Astellas Pharma Global Development, Inc.

Chief Executive Officer

Not available

IVERIC bio, Inc.

Lead Sponsor

Trials

5

Recruited

1,100+

Published Research Related to This Trial

The incidence of Stargardt disease (STGD1) was found to be between 1.67 and 1.95 cases per million per year, with a point prevalence of approximately 1 in 22,000 people in 2018, indicating it is a relatively rare condition.

Late-onset STGD1 (diagnosed at age 45 or older) has increased in recent years, making up 33% of diagnoses from 2014-2018, likely due to improved awareness, but misdiagnosis as age-related macular degeneration was common in this group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Stargardt disease: monitoring incidence and diagnostic trends in the Netherlands using a nationwide disease registry. Runhart, EH., Dhooge, P., Meester-Smoor, M., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03364153

Study Details | NCT03364153 | Zimura Compared to Sham ... The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12408533/

Real-World Experience of Geographic Atrophy Treatment ... In this study, avacincaptad pegol treatment led to a 2.03% rate of new nAMD conversion and a 3% rate of acute postinjection IOP elevation ...

3. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2023/217225Orig1s000MedR.pdf

217225Orig1s000 CLINICAL REVIEW(S) - accessdata.fda.gov Primary Objective: The objectives of this study were to evaluate the safety and efficacy of. Zimura (avacincaptad pegol) 1 mg, 2 mg, or 4 mg ...

4. clinicaltrials.eu clinicaltrials.eu/inn/avacincaptad-pegol/

Avacincaptad Pegol – Application in Therapy and Current ... Avacincaptad Pegol, also known as Zimura or Izervay, is an investigational drug being studied in clinical trials for the treatment of Geographic Atrophy (GA).

5. access.portico.org access.portico.org/Portico/show?viewFile=pdf&auId=pjbd3gm85gm

Avacincaptad pegol Avacincaptad pegol has been demonstrated to slow GA progression in AMD, but it is unclear whether inhibiting C3 or C5 will be more efficacious or safe. Clinical ...

6. clinicaltrials.astellas.com clinicaltrials.astellas.com/study/OPH2005

Zimura Compared to Sham in Patients with Autosomal ... The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal ...

7. retinalphysician.com retinalphysician.com/issues/2024/novemberdecember/the-pathology-of-stargardt-disease/

The Pathology of Stargardt Disease and Future Treatment ... Avacincaptad pegol (Izervay; Astellas) is a complement factor C5 ... Preliminary data presented to date suggest overall safety with potential ...