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

Study Overview
=================
Brief Summary
-----------------
To evaluate the safety and efficacy of Zimura™ (complement factor C5 inhibitor) compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).

Official Title
-----------------
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

Conditions
-----------------
Stargardt Disease 1

Intervention / Treatment
-----------------
* Drug: Zimura
* Other: Sham


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: At least two pathogenic mutations of ABCA4 gene confirmed by a 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 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 Zimura formulation

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1<br>Zimura dose group | Drug: Zimura<br>* Zimura Intravitreal Injection<br>* Other names: avacincaptad pegol;|
| Sham Comparator: Cohort 2<br>Sham dose group | Other: Sham<br>* Sham Intravitreal Injection<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Spectral Domain-Optical Coherence Tomography (SD-OCT) | Mean rate of change in the area of ellipsoid zone defect measured by en face SD-OCT | 18 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Zimura, ARC1905, complement factor C5 inhibitor, avacincaptad pegol, STGD1