Osprey: STK-002 Safety, Tolerability, and Exposure in ADOA patients
Research type
Research Study
Full title
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
IRAS ID
1006380
Contact name
Sherry Leonard
Contact email
Sponsor organisation
Stoke Therapeutics, Inc.
ISRCTN Number
ISRCTN41725621
Research summary
Autosomal Dominant Optic Atrophy (ADOA) is a genetic disease characterised by progressive loss in vision as a result of the breakdown of cells within the retina of the eye. This degeneration typically begins within the first decade of life, with signs generally emerging between the ages of 4 and 6. Between 65% to 90% of ADOA cases are caused by a mutation to the gene OPA1 leading to a reduction in the levels of functional OPA1 protein. This protein is critically involved in the maintenance of retinal mitochondria (small structures found within the retinal cells). Patients may experience a decrease in sharp vision, blurred vision, reduced contrast and abnormal color vision, that cannot be corrected with glasses. From early childhood, patients experience a worsening of the central part of vision that allows for proper seeing of small letters and reading. So far there is no treatment available that stops or cures the disease.
Study STK-002-OA-101 is a phase I, multi-centre, open-label study to investigate the safety, tolerability and exposure of single ascending doses of STK-002 (the study drug), an antisense oligonucleotide (ASO) in patients aged 6 to 55 who present with Autosomal Dominant Optic Atrophy (ADOA). STK-002 is an antisense oligonucleotide (ASO), a short chain of nucleotides (the basic building block of nucleic acids, like RNA) designed to increase the levels of functioning OPA1 protein in the eye, thereby halting or slowing the progression of the disease and leading to an improved quality of life.
The study will be split into two parts. Part A will assess how safe STK-002 is for participants aged ≥18 to <55 years. Once data from Part A are reviewed, they will be used to determine the dosage of STK-002 in Part B of the study, which will enrol participants ages ≥6 to <18. Participants will receive one dose of the study drug into an affected eye. The study will last approximately 48 weeks with multiple study site visits to monitor participants.REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
23/NE/0031
Date of REC Opinion
17 Mar 2023
REC opinion
Further Information Favourable Opinion