There is important news for the ADOA community. Stoke Therapeutics has started the OSPREY study, a clinical trial of STK-002, a potential treatment for Autosomal Dominant Optic Atrophy (ADOA). Participants are currently being sought for this study.
First study with STK-002 in humans
The OSPREY study is a Phase 1 clinical trial. This is the first human study of the new drug STK-002, specifically for patients with Autosomal Dominant Optic Atrophy (ADOA).
The research consists of two parts:
- Part A: adults aged 18 to 55.
- Part B: children and young people aged 6 to 18.
Participants receive a single, escalating dose of the drug. The primary goal of this phase is to investigate the safety and tolerability of STK-002. In addition, researchers are looking for initial indications of whether the treatment might have a positive effect on the disease.
How does STK-002 work?
The drug being tested is called STK-002. This is a so-called 'antisense oligonucleotide' (ASO), a short synthetic piece of genetic material that targets the RNA of the OPA1 gene. The goal is to increase the amount of functional OPA1 protein so that the function of the optic nerve can potentially be supported.
The medication is administered via an injection into the eye. Although this may sound invasive, participants are guided by specialized medical teams throughout the entire process.
The ultimate goal of the treatment is to support the function of the optic nerve and possibly preserve or improve vision. Whether this actually occurs is being investigated in a follow-up study.
Research locations
The OSPREY study is currently running in the United Kingdom, Denmark, Austria, Germany, and Italy. For all locations, if you are interested in participating in these studies, you can send an email to: ospreystudy@medpace.com
Good news for the European ADOA community: the research site in Copenhagen, Denmark, is now also welcoming international participants from across the European Union. This gives more patients and families access to participate in the study.
More information or register
Anyone who wants to know more about the research or wants to get in touch with a research team has two options.
All research locations
Visit www.ospreyclinicaltrial.comAfter selecting your language, you can contact the research team directly via 'Osprey Recruitment Team' at the bottom of the website. For Austria and Italy, you can email ospreystudy@medpace.com and indicate that you would like to be contacted by the location in Austria or Italy.
Danish research site (Copenhagen)
Send an email to ospreystudy@medpace.com and indicate that you would like to be contacted by the location in Denmark. Medpace will then share your details with the research team, who will contact you for a no-obligation discussion about the study, the procedures, potential risks, and your suitability.
Are you possibly eligible?
To ensure that the study is as safe as possible for everyone participating, the medical team uses a list of criteria.
You may be eligible if:
- you are between 18 and 55 years old (to participate in Part A of the study), or between 6 and 18 years old (to participate in Part B);
- you have the clinical diagnosis of ADOA, confirmed by a genetic test demonstrating a 'heterozygous' OPA1 gene variant;
- your residual visual acuity (BCVA), measured on a standard ETDRS letter chart, lies between 35 and 70 letters in each eye. The very first two patients in the study are an exception: they must have a score between 5 and 35 letters in each eye.
Participation is not possible when:
- certain OPA1 variants are involved, such as gain-of-function, homozygous, or compound heterozygous variants;
- extraocular symptoms are present, such as in ADOA-plus or Behr syndrome;
- other eye diseases or an increased risk of eye inflammation may influence the test results;
- there are current or previous treatments that could cause damage to the optic nerve.
Want to know more?
The official records of the study are available via the European and British clinical registers.
The Cure ADOA Foundation encourages anyone who may be eligible to educate themselves. Participation in clinical research is a personal choice, but studies such as OSPREY represent an important step toward future treatments for ADOA.
