18‐mer antisense oligonucleotide complementary to OPA1 pre-mRNA for Autosomal Dominant Optic Atrophy | Hereditary optic atrophy

Inclusion criteria

• {"criterion_text":"- 1. Patient must be ≥18 to <55 years to participate in Part A and ≥6 to <18 years to participate in Part B"}
• {"criterion_text":"- 2. Patient must have a clinical diagnosis of ADOA and have a heterozygous OPA1 gene variant confirmed at Screening by central lab genotyping"}
• {"criterion_text":"- 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

• {"criterion_text":"- Patient has a gain-of-function variant, or compound heterozygous or homozygous pathogenic or likely pathogenic variant in the OPA1 gene"}
• {"criterion_text":"- Patient has extraocular phenotypic manifestations of (syndromic) ADOA (ADOA-plus) or have Behr syndrome"}
• {"criterion_text":"- Patient has, or has a history of, any ocular condition in either eye that, in the opinion of the Investigator, could affect study parameters"}
• {"criterion_text":"- Patient is considered to be at risk for uveitis or ocular infection during the study period"}
• {"criterion_text":"- Patient is taking, or has taken at any time, any medication or treatment that can or might cause an optic neuropathy"}

Primary endpoints

• {"endpoint_text":"- Safety variables for analysis","definition_or_measurement_approach":""}
• {"endpoint_text":"- The exposure of STK-002 in serum will be determined using Pharmacokinetic (PK) parameters","definition_or_measurement_approach":"Determined using Pharmacokinetic (PK) parameters"}

Secondary endpoints

• {"endpoint_text":"- Peripapillary retinal nerve fiber layer (RNFL) and macular ganglion cell layer (GCL, or ganglion cell complex) thicknesses as assessed by optical coherence tomography (OCT)","definition_or_measurement_approach":"Assessed by optical coherence tomography (OCT)"}
• {"endpoint_text":"- Best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) optotypes","definition_or_measurement_approach":"Measured as BCVA using ETDRS optotypes"}
• {"endpoint_text":"- Contrast sensitivity as assessed by low contrast BCVA (at 25%, 5% and 2.5% contrast levels)","definition_or_measurement_approach":"Assessed by low contrast BCVA at 25%, 5% and 2.5% contrast levels"}
• {"endpoint_text":"- Visual field (as assessed by automated, static perimetry [10-2 and 24-2 Swedish Interactive Threshold Algorithm (SITA) FAST])","definition_or_measurement_approach":"Assessed by automated static perimetry using 10-2 and 24-2 SITA FAST"}
• {"endpoint_text":"- Electrical activity of the retina as assessed by phototopic negative response (PhNR) ERG (if available)","definition_or_measurement_approach":"Assessed by PhNR ERG (if available)"}
• {"endpoint_text":"- Continuous text reading acuity as assessed by MNREAD Acuity Charts","definition_or_measurement_approach":"Assessed using MNREAD Acuity Charts"}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Measured using NEI-VFQ-25, IVI-C, and EQ-5D/EQ-5D-Y questionnaires"}

Germany

Earliest CTIS Part Ii Submission Date

05-04-2024

Latest Decision Or Authorization Date

14-10-2025

Processing Time Days

557

Number Of Sites

2

Number Of Participants

14

Denmark

Earliest CTIS Part Ii Submission Date

01-04-2024

Latest Decision Or Authorization Date

13-10-2025

Processing Time Days

560

Number Of Sites

1

Number Of Participants

10

Austria

Earliest CTIS Part Ii Submission Date

02-04-2024

Latest Decision Or Authorization Date

08-12-2025

Processing Time Days

615

Number Of Sites

1

Number Of Participants

9

Italy

Earliest CTIS Part Ii Submission Date

29-07-2024

Latest Decision Or Authorization Date

12-12-2025

Processing Time Days

501

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Stoke Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Iqvia Holdings Inc.

Responsibilities

Pharmacovigilance

Name

Medpace Finland Oy

Responsibilities

Patient Travel; Patient Questionnaires and Vision Charts; other site/patient support (codes 1,3,5 present in duties list)

Name

PPD Global Central Labs

Responsibilities

Central laboratory services (duty code 4)

Name

DARC

Responsibilities

Central Imaging

Name

Medidata Solutions Inc.

Responsibilities

Data capture/clinical trial systems (duty code 7)

Third parties

• {"country":"United States","full_name":"Iqvia Holdings Inc.","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
• {"country":"Finland","full_name":"Blueprint Genetics","duties_or_roles":"code:4","organisation_type":"Industry"}
• {"country":"United States","full_name":"DARC","duties_or_roles":"Central Imaging","organisation_type":"Industry"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"1; Patient Travel; Patient Questionnaires and Vision Charts; 3; 5","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}