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
Autosomal dominant optic atrophy
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 visit 3 (Day 1) of their participation in the study. Participants in part A of the study will be assigned to one of four groups. Each group will receive a different dose of the study drug starting at a safe, low dose in group 1, which was calculated by data collected through pre-clinical studies, and escalating to higher doses in groups 2, 3 and 4. Group 1 will be the first to receive the study drug and data collected from participants will be reviewed by a Safety Monitoring Committee (SMC). The SMC will use this data to determine whether the dose can be escalated and given to the next group. This process will be repeated for all groups. An optional two additional groups may be added to Part A, with the dose in these groups escalated further. Administration of the study drug will take approximately 5-10 minutes. However, the total time for administration procedure may take up to 2 hours if patients require sedation. Data from Part A of the study will be reviewed by the SMC to determine a safe dose of the study drug to be given to participants in Part B of the study. In Part B, the first paediatric participant (sentinel) to receive the study drug will be ≥12 and <18 years of age. The SMC will meet again after dosage to determine whether to continue to dose, and for dose escalation/de-escalation for additional groups in Part B. There are currently 10 planned visits to the research site and via one planned telephone call to follow up with participants. Follow-up will last approximately up to 50 weeks after study drug administration, to evaluate the safety and tolerability of the study drug. _____ Previous interventions: 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 ey
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
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