Zimura for Stargardt Disease

Recruiting at 49 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

121 Participants Needed

Trial Summary

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.

How does the drug Zimura differ from other treatments for Stargardt Disease?

Zimura is unique because it targets the C5 component of the complement system, which is part of the body's immune response, potentially slowing the progression of Stargardt Disease. Unlike other treatments, it specifically inhibits this part of the immune system, and there are currently no approved treatments for this condition. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Research Team

Medical Director

Principal Investigator

Astellas Pharma Global Development, Inc.

Eligibility Criteria

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 macular atrophy not caused by STGD1 in either eye.

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.

Timeline

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Effects of Photo-Biomodulation in Stargardt Disease. [2022]

2. Spain pubmed.ncbi.nlm.nih.gov

Stargardt Disease: a clinical case report of two sisters with different clinical development. [2021]

3. Spain pubmed.ncbi.nlm.nih.gov

Stargardt disease: A clinical case report of two sisters with different clinical development. [2022]

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

Stargardt's disease: a review of the literature. [2005]

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

Stargardt disease: monitoring incidence and diagnostic trends in the Netherlands using a nationwide disease registry. [2022]