Autosomal dominant optic atrophy Eye Diseases
Conditions
Interventions
Current interventions as of 08/03/2024:
STK-002-OA-101 is an open-label study in which a participant will receive a single dose of the study drug STK-002, via an injection to the affected eye, at vi
Sponsors
Stoke Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
6 Years to 60 Years
Inclusion criteria
Inclusion criteria: Current key inclusion criteria as of 10/07/2026: 1. Patient must be =18 to <55 years to participate in Part A and =6 to <18 years to participate in Part B 2. Patient must have a clinical diagnosis of ADOA and have a heterozygous OPA1 gene variant confirmed at Screening by central lab genotyping 3. Patient must have a BCVA EDTRS letter score of =35 and =70 in the intended treated eye, with the exception of the first two patients in Cohort 1 of Part A who must have a BCVA ETDRS letter score =5 and =35 in the intended treated eye 4. Patient’s screening/baseline full field electroretinogram (ffERG) and optical coherence tomography (OCT) images are acceptable prior to dosing _____ Previous key inclusion criteria as of 29/04/2026: 1. Patient must be =18 to <55 years to participate in Part A and =6 to <18 years to participate in Part B 2. Patient must have a clinical diagnosis of ADOA and have a heterozygous OPA1 gene variant confirmed at Screening by central lab genotyping 3. Patient must have a BCVA EDTRS letter score of =35 and =70 with each eye individually, with the exception of the first two patients in Cohort 1 of Part A who must have a BCVA ETDRS letter score =5 and =35 in each eye 4. Patient’s screening/baseline full field electroretinogram (ffERG) and optical coherence tomography (OCT) images are acceptable prior to dosing _____ Previous key inclusion criteria: 1. Patient must be =18 to <55 years to participate in Part A and =6 to <18 years to participate in Part B 2. Patient must have a clinical diagnosis of ADOA and have a heterozygous OPA1 gene variant confirmed at Screening by central lab genotyping 3. Patient must have a BCVA EDTRS letter score of =35 and =70 with each eye individually, with the exception of the first two patients in Cohort 1 of Part A who must have a BCVA ETDRS letter score =5 and =35 in each eye
Exclusion criteria
Exclusion criteria: 1. Patient has a gain-of-function variant, or compound heterozygous or homozygous pathogenic or likely pathogenic variant in the OPA1 gene 2. Patient has extraocular phenotypic manifestations of (syndromic) ADOA (ADOA-plus) or has Behr syndrome 3. Patient has or has a history of, any ocular condition in either eye that, in the opinion of the Investigator, could affect study parameters 4. Patient is considered to be at risk for uveitis or ocular infection during the study period 5. Patient is taking or has taken at any time, any medication or treatment that can or might cause an optic neuropathy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Current primary outcome measure as of 08/03/2024: Evaluation of these endpoints will occur at up to 48 weeks (+/- 2 weeks) after Study Drug Administration: 1. Safety variables for analysis include: 1.1. The incidence, type, and severity of adverse events (AEs) in the treated eye throughout the study 1.2. Changes in vital signs at day 1, 2, 8, 29 and 337 1.3. Laboratory parameters in up to 7 of the 10 visits 1.4. Age-Related Eye Disease Study (AREDS) Clinical Lens Grading System scores at day 2, 8, 29, 85, 169, 253 and 337 1.5. Assessment of inflammation (Standardization of Uveitis Nomenclature [SUN] Anterior Chamber Cell score and NEI Vitreous Haze scores) at baseline, day 8, 29, 85, 169, 253 and 337 1.6. Corneal epithelial grading using Efron corneal grading scale at baseline, day 2, 8, 29, 85, 169, 253, and 337 1.7. Physical examination at day 2, 8, 29 and 337 1.8. Immunogenicity parameters at baseline, day 29, 85, 169 and 337 1.9. Retinal Toxicity assessed by full-field electroretinogram (ERG), retinal thinning by optical coherence tomography (OCT) at baseline, day 29, 85, 169, 253, and 337 2. The exposure of STK-002 in serum will be determined measured pre-dose, at 1, 2, 4, 6, 24 hours post dose and on days 8, 29, 85, 169 and 337. Pharmacokinetic (PK) parameters may include: 2.1. Maximum observed concentration (Cmax) 2.2. Time to maximum observed concentration (Tmax) 2.3. Fold change in Cmax compared with dose level _____ Previous primary outcome measure: Evaluation of these endpoints will occur at up to 48 weeks (+/- 2 weeks) after Study Drug Administration: 1. Safety variables for analysis include: 1.1. The incidence, type, and severity of adverse events (AEs) throughout the study 1.2. Changes in vital signs at day 1, 2, 8, 29 and 337 1.3. Laboratory parameters in up to 7 of the 10 visits 1.4. Age-Related Eye Disease Study (AREDS) Clinical Lens Grading System scores at day 2, 8, 29, 85, 169, 253 and 337 1.5. Assessment of inflammation (Standardization of | — |
Secondary
| Measure | Time frame |
|---|---|
| Current key secondary outcome(s) as of 29/04/2026: Secondary endpoints for STK-002 evaluated by dose level and change from baseline at each visit are assessed at up to 48 weeks (+/- 2 weeks) after Study Drug Administration: 1. Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT) at baseline, day 29, 85, 169, 253 and 337 2. Best-corrected visual acuity (BCVA) measured using Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes at screening, and days 8, 29, 85, 169, 253 and 337 3. Contrast sensitivity assessed by low contrast BCVA (at 25% and 5% contrast levels) at baseline, and days 29, 85, 169, 253 and 337 4. Visual field as assessed by automated, static perimetry [10-2 Swedish Interactive Threshold Algorithm (SITA) FAST] and 24-2 Short Wavelength Automated Perimetry [SWAP] at baseline, and days 29, 85, 169, 253 and 337 5. Electrical activity of the retina as assessed by photopic negative response (PhNR) ERG, at baseline, and days 29, 85, 169, 253 and 337 6. Continuous text reading acuity as assessed by MNREAD Acuity Charts at baseline, and days 29, 85, 169, 253 and 337 7. Quality of life 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 at baseline, and days 29, 85 and 337 _____ Previous secondary outcome measures as of 08/03/2024: Secondary endpoints for STK-002 evaluated by dose level and change from baseline at each visit are assessed at up to 48 weeks (+/- 2 weeks) after Study Drug Administration: 1. Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT) at baseline, day 29, 85, 169, 253 and 337 2. Best-corrected visual acuity (BCVA) measured usi | — |
Countries
Austria, Denmark, England, Germany, Italy, United Kingdom, Wales
Outcome results
None listed