The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation. Subjects will receive QR-110 in one eye every 3 months, for a maximum of 4 doses. Up to 3 dose levels of QR-110 will be evaluated.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
11
RNA antisense oligonucleotide for intravitreal injection
University of Iowa
Iowa City, Iowa, United States
Scheie Eye Institute, University of Pennsylvania
Philadelphia, Pennsylvania, United States
Ghent University Hospital and Ghent University
Ghent, Belgium
Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes
Time frame: 1 year
Frequency and Severity of Non-ocular Adverse Events
Time frame: 1 year
Change in Best-corrected Visual Acuity (BCVA)
Time frame: 1 year
Change in Full-field Stimulus Test (FST)
Average Red Light Score
Time frame: 1 year
Change in Full-field Stimulus Test (FST)
Average Blue Light Score
Time frame: 1 year
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