Zimura for Stargardt Disease

Recruiting in Palo Alto (17 mi)

+49 other locations

Age: 18 - 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Astellas Pharma Global Development, Inc.

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Trial Summary

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.

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.

+9 more

Participant Groups

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.

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.