Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease

The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).

Inclusion Criteria:

• At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
• Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Exclusion Criteria:

• Macular atrophy secondary to any condition other than STGD1 in either eye
• Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
• Participation in an interventional study of a vitamin A derivative </= 3 months prior to screening
• Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
• Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
• Diabetes mellitus
• Hemoglobin A1c (HbA1c) value of >/=6.5%
• Stroke within 12 months of trial entry
• Any major surgical procedure within one month of trial entry or anticipated during the trial
• Any treatment with an investigational agent in the past 60 days for any condition
• Women who are pregnant or nursing
• Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation