Osprey: An Open-label Study to Investigate the Safety, Tolerability, and Exposure of Single Ascending Doses of the Antisense Oligonucleotide STK-002 in Patients with Autosomal Dominant Optic Atrophy

Conditions

Autosomal Dominant Optic Atrophy

Brief summary

Safety variables for analysis, The exposure of STK-002 in serum will be determined using Pharmacokinetic (PK) parameters

Detailed description

Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT), Best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes, Contrast sensitivity as assessed by low contrast BCVA (at 25%, 5% and 2.5% contrast levels), Visual field (as assessed by automated, static perimetry [10-2 and 24-2 Swedish Interactive Threshold Algorithm (SITA) FAST]), Electrical activity of the retina as assessed by phototopic negative response (PhNR) ERG (if available), Continuous text reading acuity as assessed by MNREAD Acuity Charts, Quality of life as measured by scores on the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25), Impact of Vision Impairment for Children (IVI-C), and the European Quality of Life-5 Dimensions (EQ-5D)/EQ-5D-Y questionnaire

Related papers

No related papers found yet.

Interventions

DRUGSTK-002

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Safety variables for analysis, The exposure of STK-002 in serum will be determined using Pharmacokinetic (PK) parameters	—

Secondary

Measure	Time frame
Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT), Best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes, Contrast sensitivity as assessed by low contrast BCVA (at 25%, 5% and 2.5% contrast levels), Visual field (as assessed by automated, static perimetry [10-2 and 24-2 Swedish Interactive Threshold Algorithm (SITA) FAST]), Electrical activity of the retina as assessed by phototopic negative response (PhNR) ERG (if available), Continuous text reading acuity as assessed by MNREAD Acuity Charts, Quality of life as measured by scores on the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25), Impact of Vision Impairment for Children (IVI-C), and the European Quality of Life-5 Dimensions (EQ-5D)/EQ-5D-Y questionnaire	—

Measure

Time frame

Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT), Best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes, Contrast sensitivity as assessed by low contrast BCVA (at 25%, 5% and 2.5% contrast levels), Visual field (as assessed by automated, static perimetry [10-2 and 24-2 Swedish Interactive Threshold Algorithm (SITA) FAST]), Electrical activity of the retina as assessed by phototopic negative response (PhNR) ERG (if available), Continuous text reading acuity as assessed by MNREAD Acuity Charts, Quality of life as measured by scores on the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25), Impact of Vision Impairment for Children (IVI-C), and the European Quality of Life-5 Dimensions (EQ-5D)/EQ-5D-Y questionnaire

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Countries

Austria, Denmark, Germany, Italy

Outcome results

None listed