A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
A Phase 1 Open-Label, Multiple Ascending Dose Study of VP-001 in Participants with Confirmed PRPF31 Mutation- Associated Retinal Dystrophy
University of Florida Health
Jacksonville, Florida, United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States
Oregon Health and Science University - Casey Eye Institute
Portland, Oregon, United States
Retina Foundation of the Southwest
Dallas, Texas, United States
The incidence, severity, and relatedness of treatment-emergent ocular adverse events and treatment-emergent serious adverse events
Time frame: over a 4-week post-dose period
The incidence, severity, and relatedness of treatment-emergent ocular adverse events
Time frame: over a 52-week period
Adverse Events and Treatment Emergent serious adverse events (TESAEs) in the fellow eye eye
Time frame: over a 4-week post-dose time period
Adverse Events and Treatment Emergent serious adverse events (TESAEs) in the fellow eye eye
Time frame: over a 52-week period
Incidence, severity, and relatedness of non-ocular Treatment Emergent adverse events (TEAEs)
Time frame: over a 4-week post-dose time period
Incidence, severity, and relatedness of non-ocular Treatment Emergent adverse events (TEAEs)
Time frame: over a 52-week period
BCVA letter score using ETDRS charts
Time frame: over a 52-week period
Change in lowest passing light level using Ora- VNC™ mobility test
Time frame: over a 52-week period
Low luminance visual acuity (LLVA) letter score
Time frame: over a 52-week period
Visual field sensitivity as measured by static perimetry with topographic analysis (Hill of Vision)
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Baylor College of Medicine- Alkek Eye Center
Houston, Texas, United States
Time frame: over a 52-week period
Mean retinal sensitivity as measured by fundusguided microperimetry
Time frame: over a 52-week period
Visual fields as measured by kinetic perimetry utilizing I4e, II4e and V4e stimuli
Time frame: over a 52-week period
Rod-and cone-mediated retinal function as measured by white, red and blue FST
Time frame: over a 52-week period
Retinal thickness on SD-OCT, including retinal thickness in each ETDRS subfield and EZ area and volume
Time frame: over a 52-week period
Retinal function using full-field electroretinography (ERG)
Time frame: over a 52-week period
Area of hypo-autofluorescence captured by FAF
Time frame: over a 52-week period
Abnormalities captured by wide-field fundus photography
Time frame: over a 52-week period