30 closest matches · ranked by relevance
Leber Congenital Amaurosis
Primary: Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event — 0; 0; 1; 0 Participants
Leber Congenital Amaurosis
Primary: Number of Participants Experiencing Ocular or Non-ocular Adverse Events — 6; 6 participants
Leber's Congenital Amaurosis
Primary: Frequency and Severity of Ocular Adverse Events in the Treatment and Contralateral Eyes — 6; 5 Participants
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Achromatopsia
Primary: Number of Participants Meeting the Primary Outcome Defined as Any of the Below Events Occurring During the 6 Weeks Following Administration, at Least Possibly Related to…
Leber's Hereditary Optic Neuropathy (LHON)
Primary: In Eyes With VA Assessment Made ≤1 Year After Onset of Symptoms: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA (Measured by Change in ETDRS Letters)…
Leber Hereditary Optic Neuropathy
Primary: The Best Corrected Visual Acuity(BCVA) — 1.40; 1.69; 1.36; 1.58 logMAR
Leber's Hereditary Optic Neuropathy
Primary: Double Masked Period: Incidence of Ocular TEAEs — 3; 8; 1; 3 eyes
Retinitis Pigmentosa · X-linked Genetic Diseases
Primary: Rate of LOSS of 31 Hertz Cone Electroretinographic Function — 0.028; 0.022 log microvolts/year — p=0.30
Leber's Hereditary Optic Neuropathy
Primary: Number of Treatment Related Adverse Events — 1; 1; 1; 5 Participants
Retinitis Pigmentosa
Primary: ETDRS Visual Acuity (High Contrast) — -0.02; 0.11 log unit
Age-Related Macular Degeneration
Primary: Phase I: Percentage of Participants With Adverse Events — 11; 10; 10 Participants — p=1.00
Albinism
Primary: Improved Vision — 0.67; 0.55; 0.53 logMAR
Optic, Atrophy, Hereditary, Leber
Primary: Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48 — 0.380; 0.392 LogMAR — p=0.8890
Age-related Macular Degeneration
Primary: Change in Best Corrected Visual Acuity — 6.0; 4.6 Change in letters (BCVA)
Retinitis Pigmentosa · Usher Syndrome Type 2 · Usher Syndrome Type 3
Primary: Mean Change in Cone Spacing in Arcminutes (Z Score) of 2 Baseline Values Were Compared With Measurements Obtained at Post-op Month 36 — 1.13; 0.85 Difference in change…
Eye Disease · Achromatopsia
Primary: Number of Adverse Events at Six Months Post-Implantation — 10 Adverse Events
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 Participants
Amblyopia
Primary: Distribution of Amblyopic Eye Visual Acuity Change From Baseline — 0; 0; 2; 1 participants
Retinopathy of Prematurity
Primary: Changes in mRNA Expression in Blood of STAT3, PPAR-ɣ, and STC-1 at T0 — 4.19; 7.58; 6.43; 11.32 delta Ct (amplification cycles) — p=0.22
Retinitis Pigmentosa
Primary: Central Visual Field (vf) Change Assessed Using the 30-2 Program of the Humphrey Field Analyzer (HFA). — -51.5; -49.6 annual change in db vf sensitivity — p=0.66
Optic, Atrophy, Hereditary, Leber
Primary: Change From Baseline in ETDRS Visual Acuity (Quantitative Score) at Week 48 — -0.219; -0.211 LogMAR — p=0.8783
Age-related Macular Degeneration
Primary: Height (Amplitude) of Multifocal ERG Signal — 2.859 nanovolt/degree^2 — p=<0.05
Macular Degeneration · Wet Macular Degeneration
Primary: Anatomic Response
Age-related Macular Degeneration
Primary: Change in Best Corrected Visual Acuity by Early Treatment Diabetic Retinopathy Study Chart Visual Scale Testing — 4.5 Change in letters (BCVA)
Retinoschisis · X-Linked
Primary: Adverse Events (AEs) Affecting Ocular Function That Differ Clinically From Those Expected in the Normal Course of Progression of XLRS — 3; 3; 12; 6 events
Macular Degeneration
Primary: Mean Change in Visual Acuity — 15.0; 5.6 letters — p=0.02
Albinism · Vision Loss
Primary: Absolute Mean Change in Iris Pigmentation on an 8-point Iris Transillumination Scale at 12 Months as Compared to Baseline. Participants Left and Right Eyes Will be…
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Leber Hereditary Optic Neuropathy
Primary: Change From Baseline of the Best Corrected Visual Acuity (BCVA) Reported With Log of the Minimal Angle of Resolution (LogMAR) at 1.5 Years Post-treatment, in the Second…