Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1) (NCT03364153) | Clinical Trial Compass
CompletedPhase 2
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
United States121 participantsStarted 2018-01-12
Plain-language 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).
Who can participate
Age range18 Years – 60 Years
SexALL
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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
What they're measuring
1
Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18