Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Study PQ-110-002 will provide long-term safety, tolerability, pharmacokinetic (PK), and efficacy data of QR-110.
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study for continued dosing if available data support current and/or future benefits for the subject. Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current and/or future benefits for the subject. The Investigator, in consultation and agreement with the Medical Monitor, will decide on enrollment of each individual subject, as well as on dosing of the first treated eye and treatment initiation of the contralateral eye. Continued subject treatment in this study is desirable, but cannot be guaranteed, since it will depend on the risks and benefit of further treatment on a case-by-case basis, as discussed and agreed upon with the Medical Monitor. The contralateral eye and the first treated eye will be injected 3 months apart. The injection interval of 3 months between both eyes will limit burden for the subjects, with a 3 month-visit frequency during the course of the study. This between-eye interval could be adapted if safety data are supportive, and for logistic reasons, and in agreement with the Medical Monitor. The same safety monitoring protocol and efficacy assessments will apply to both eyes. QR-110 will be administered via intravitreal (IVT) injection.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
First treated eye: maintenance dose every 6 months, intravitreal administration Contralateral eye: loading dose followed by maintenance dose, every 6 months, intravitreal administration
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 of Ocular AEs
Frequency of ocular adverse events (AEs)
Time frame: 24 months
Frequency of Non-ocular AEs
Frequency of non-ocular AEs
Time frame: 24 months
Change in BCVA in First Treated Eye
Change in Best Corrected Visual Acuity (BCVA) in First Treated Eye
Time frame: 24 months
Change in Mobility Course Score
Change in Mobility course score
Time frame: 24 months
Change in Photoreceptor Outer Segment Layer Thickness
Change in photoreceptor outer segment layer thickness by Optical Coherence Tomography (OCT)
Time frame: 24 months
Change in OCI
Change in Oculomotor Instability (OCI)
Time frame: 24 months
Change in FST Blue
Change in Full-Field Stimulus Testing (FST) - blue stimuli
Time frame: 24 months
Change in FST Red
Change in Full-Field Stimulus Testing (FST) - red stimuli
Time frame: 24 months
Change in VFQ-25
Change in Visual Function Questionnaire-25 (VFQ-25) score (adult subjects)
Time frame: 24 months
Change in CVAQ
Change in Cardiff Visual Ability Questionnaire for Children (CVAQC) score (pediatric subjects)
Time frame: 24 months
Change in PLR
Change in Pupillary Light Reflex (PLR) (latency and amplitude)
Time frame: 24 months
Change in NIRAF
Change in Near Infrared AutoFluorescence (NIRAF)
Time frame: 24 months
Change in BCVA in Treated Contralateral Eye
Change in Best Corrected Visual Acuity (BCVA) in Treated Contralateral Eye
Time frame: 24 months
Change in BCVA in Non-Treated Contralateral Eye
Change in Best Corrected Visual Acuity (BCVA) in Non-Treated Contralateral Eye
Time frame: 24 months
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