Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
Purpose
To evaluate the safety and efficacy of Zimura™ (complement factor C5 inhibitor) compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).
Condition
- Stargardt Disease 1
Eligibility
- Eligible Ages
- Between 18 Years and 60 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Cohort 1 |
Zimura dose group |
|
|
Sham Comparator Cohort 2 |
Sham dose group |
|
Recruiting Locations
More Details
- NCT ID
- NCT03364153
- Status
- Active, not recruiting
- Sponsor
- IVERIC bio, Inc.