Phase 2
N=121
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
Stargardt's Macular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT03364153 ↗Enrolled (actual)
121
Serious AEs
4.2%
Results posted
Mar 2026
Primary outcome: Primary: Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18 — 0.6383; 0.6644 mm^2/18 months — p=0.8669
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- avacincaptad pegol (Drug); Sham (Drug)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Astellas Pharma Global Development, Inc.
- Primary completion
- Mar 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18 |
0.6383; 0.6644 | 0.8669 |
| SECONDARY Change in Best Corrected Visual Acuity (BCVA) Using Early Treatment Diabetic Retinopathy Study (ETDRS) Letters From Baseline at Month 18 |
-0.1326; -1.3358 | 0.4745 |
| SECONDARY Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18 |
-1.1604; -1.9184 | 0.5145 |
| SECONDARY Number of Participants With Adverse Events (AEs) |
53; 45 | — |
Summary
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).
Eligibility Criteria
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 /=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
Data sourced from ClinicalTrials.gov (NCT03364153). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.