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
Retinitis Pigmentosa
Primary: ETDRS Visual Acuity (High Contrast) — -0.02; 0.11 log unit
Leber's Hereditary Optic Neuropathy
Primary: Number of Treatment Related Adverse Events — 1; 1; 1; 5 Participants
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
Albinism
Primary: Improved Vision — 0.67; 0.55; 0.53 logMAR
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
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: Phase I: Percentage of Participants With Adverse Events — 11; 10; 10 Participants — p=1.00
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
Amblyopia
Primary: Distribution of Amblyopic Eye Visual Acuity Change From Baseline — 0; 0; 2; 1 participants
Age-related Macular Degeneration
Primary: Change in Best Corrected Visual Acuity — 6.0; 4.6 Change in letters (BCVA)
Cerebrotendinous Xanthomatosis (CTX)
Primary: Number of Participants Who Had Positive Genetic Test Results — 4 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
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
Lysosomal Acid Lipase Deficiency
Primary: Percentage Of Participants Achieving Alanine Aminotransferase Normalization — 31; 7; 56; 37 percentage of participants — p=0.0271
Age-related Macular Degeneration
Primary: Height (Amplitude) of Multifocal ERG Signal — 2.859 nanovolt/degree^2 — p=<0.05
Niemann-Pick Disease, Type C
Primary: Efficacy of Adrabetadex (VTS-270) to Reduce Plasma Levels of a Conjugated Bile Acid, Known as 5α-cholanic Acid-3β, 5α, 6β-triol N-(Carboxymethyl)-Amide — 32.6; 87.9 ng/ml
Infantile Neronal Ceroid Lipofuscinosis
Primary: Change in Cellular Granular Osmiophilic Deposits (GRODs) in Electron Micrographs of Peripheral White Blood Cells. — -0.3317 Average number of GRODs
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)
Cholesterol Ester Storage Disease(CESD) · Lysosomal Acid Lipase Deficiency · LAL-Deficiency
Primary: Number Of Participants Reporting TEAEs And Infusion-Related Reactions (IRRs) — 1; 3; 3; 0 participants
Macular Degeneration · Wet Macular Degeneration
Primary: Anatomic Response
Geographic Atrophy · Age-related Macular Degeneration
Primary: Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505 — 1.95; 1.58 mm^2