The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Funding Source- FDA Office of Orphan Products Development (OOPD).
Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures.
Are willing to adhere to the clinical protocol and able to perform testing procedures.
In part A participants must be 13 years of age or older at consent, for Part B, participants must be 4 years of age or older at consent with the ability to conduct the MLoMT.
Carry disease-causing biallelic LCA5 gene mutations determined by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory (historic testing up to 15 years from date of consent can be considered).
Women who are pregnant or individuals (women of childbearing potential and men) unwilling to use effective contraception for the duration of the study, including barrier methods for the first year after investigational product (IP) administration (Part A only).
Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery. This includes individuals who are immunocompromised.
History of intraocular surgery for either eye within 6 months prior to planned IP administration (Part A only).
Have previously received gene therapy.
Parallel
AAV8.hLCA5
Biological/VaccineAdeno-associated virus vector expressing human LCA5 gene
Incidence of Dose Limiting Toxicities
Number of DLT events
Number of adverse events related to OPGx-001
Number of AEs related to IP
Incidence of adverse events related to OPGx-001
Incidence of AEs related to IP
Severity of adverse events related to OPGx-001
Severity of AEs related to IP
Change from baseline over time in Multi-luminance Orientation and Mobility Test
Number of objects identified
Change from baseline over time in Dark-adapted full-field sensitivity testing
FST
Change from baseline over time in best corrected visual acuity (BCVA)
Measured using LogMAR scale
Change from baseline over time in visual functioning questionnaire
Assessed by the Modified National Eye Institute Visual Functioning Questionnaire
Microperimetry
Mean sensitivity, in dB
Kinetic perimetry
Field extent, in degrees
Virtual Reality Orientation and Mobility Test (VR-O&M)
Performance over a range of light levels
Frequency doubling technology (FDT) perimetry (sFDT-CA)
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Opus Genetics, Inc
Lead sponsor
University of Pennsylvania
Collaborator
