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).
This is a non-randomized, open-label, phase 1/2 dose-escalation study evaluating untreated patients for 6 months and with three doses of OPGx-001 for the treatment of LCA5-IRD. Enrollment will begin with a low-dose of OPGx-001 delivered via single, unilateral subretinal injection (Cohort 1) and proceed to an intermediate dose (Cohort 2) and subsequent high dose (Cohort 3). Escalation to each next cohort will proceed only after review of all data and upon recommendation by an independent data monitoring committee (IDMC). Concurrently, 16 untreated patients will be assessed for 6 months prior to treatment to study the natural course of LCA5-IRD.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
22
Adeno-associated virus vector expressing human LCA5 gene
University of Pennsylvania Perelman School of Medicine
Philadelphia, Pennsylvania, United States
RECRUITINGRetina Foundation of the Southwest
Dallas, Texas, United States
RECRUITINGIncidence of Dose Limiting Toxicities
Number of DLT events
Time frame: 2 years
Number of adverse events related to OPGx-001
Number of AEs related to IP
Time frame: 2 years
Incidence of adverse events related to OPGx-001
Incidence of AEs related to IP
Time frame: 2 years
Severity of adverse events related to OPGx-001
Severity of AEs related to IP
Time frame: 2 years
Number of procedure-related adverse events
Number of AEs related to IP administration
Time frame: 2 years
Incidence of procedure-related adverse events
Incidence of AEs related to IP administration
Time frame: 2 years
Severity of procedure-related adverse events
Severity of AEs related to IP administration
Time frame: 2 years
Assessment of cross-sectional spectral domain optical coherence tomography images
Qualitative Assessment of SD-OCT image
Time frame: 2 years
Assessment of Natural course of LCA5-IRD
Change from baseline in MLoMT
Time frame: 6 months
Assessment of Natural course of LCA5-IRD
Jasminder Soto
CONTACT
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Change from baseline in FST
Time frame: 6 months
Assessment of Natural course of LCA5-IRD
Change from baseline in BCVA
Time frame: 6 months
Assessment of Natural course of LCA5-IRD
Change from baseline in FCP/Microperimetry
Time frame: 6 months
Assessment of Natural course of LCA5-IRD
Change from baseline in SD-OCT
Time frame: 6 months
Assessment of Natural course of LCA5-IRD
Change from baseline in pupillometry
Time frame: 6 months
Assessment of Natural course of LCA5-IRD
Number of adverse events
Time frame: 6 months
Change from baseline over time in Multi-luminance Orientation and Mobility Test
Number of objects identified
Time frame: 2 years
Change from baseline over time in Dark-adapted full-field sensitivity testing
FST
Time frame: 2 years
Change from baseline over time in best corrected visual acuity (BCVA)
Measured using LogMAR scale
Time frame: 2 years
Change from baseline over time in visual functioning questionnaire
Assessed by the Modified National Eye Institute Visual Functioning Questionnaire
Time frame: 1 year