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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
121
Intravitreal Injection
Intravitreal Injection
Rate of change in the area of ellipsoid zone defect
The area of ellipsoid zone defect will be measured by en face spectral domain-optical coherence tomography (SD-OCT).
Time frame: Baseline up to 18 Months
Change in best corrected visual acuity
Best corrected visual acuity (BCVA) will be measured by Early Treatment Diabetic Retinopathy Study \[ETDRS\] letters chart.
Time frame: Baseline up to 18 Months
Change in photopic and/or mesopic macular sensitivity
The photopic and/or mesopic macular sensitivity will be measured by microperimetry (optional assessment).
Time frame: Baseline up to 18 Months
Rate of change in the area of atrophic lesion (definite decrease in autofluorescence)
The definite decrease in autofluorescence (DDAF) will be measured by fundus autofluorescence (FAF).
Time frame: Baseline up to 18 Months
Rate of change in the thickness of the outer nuclear layer
The thickness of outer nuclear layer will be measured by a horizontal scan through the foveal center using SD-OCT.
Time frame: Baseline up to 18 Months
Time to persistent vision loss
Vision loss is defined as BCVA loss \>/= 10, 15 or 20 letters from Baseline at two or more consecutive visits through Month 18.
Time frame: Baseline up to 18 Months
Emergence of at least one new atrophic lesion (DDAF)
The DDAF will be measured by FAF.
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Retinal Research Institute
Phoenix, Arizona, United States
Jules Stein Eye Institute/ David Geffen School of Medicine
Los Angeles, California, United States
VitreoRetinal Associates
Gainesville, Florida, United States
Retina Specialty Institute
Pensacola, Florida, United States
Wilmer Eye Institute, Johns Hopkins
Baltimore, Maryland, United States
Ophthalmic Consultants of Boston
Boston, Massachusetts, United States
University of Michigan/Kellogg Eye Center
Ann Arbor, Michigan, United States
The Retina Center
Minneapolis, Minnesota, United States
Retina Center of NJ, LLC.
Bloomfield, New Jersey, United States
Casey Eye Institute/Oregon Health & Science University
Portland, Oregon, United States
...and 31 more locations
Time frame: Up to 18 Months
Number of participants with Adverse Events (AEs)
An AE is defined as any untoward medical occurrence in a participant including unfavorable and unintended signs, symptoms or disease temporally associated with the use of a medicinal product and which does not necessarily have to have a causal relationship to this treatment. AEs include illnesses with onset during the trial, or exacerbations of pre-existing illnesses. Exacerbation of pre-existing illness is defined as a significant increase in the severity of the illness as compared to the start of the trial and should be considered when a patient requires new or additional treatment for that illness.
Time frame: Up to 18 Months
Number of participants with vital sign abnormalities and/or AEs
Number of participants with potentially clinically significant vital sign values.
Time frame: Up to 18 Months
Number of participants with ophthalmic abnormalities and/or AEs
Number of participants with potentially clinically significant ophthalmic variables.
Time frame: Up to 18 Months
Number of participants with 12-Lead electrocardiogram (ECG) abnormalities and/or AEs
Number of participants with potentially clinically significant 12-Lead ECG values.
Time frame: Up to 18 Months
Number of participants with laboratory value abnormalities and/or AEs
Number of participants with potentially clinically significant laboratory values.
Time frame: Up to 18 Months