Ongoing Clinical Trials for Hereditary Optic Atrophy
There is currently one ongoing clinical trial investigating a new treatment for hereditary optic atrophy, specifically for patients with autosomal dominant optic atrophy (ADOA). This trial is testing an experimental medication called STK-002, delivered through injections into the eye, and is being conducted across several European countries including Austria, Germany, Italy, and Denmark. (Also known as: Autosomal Dominant Optic Atrophy)
Clinical trial locations
- Austria
- Denmark
- Germany
- Italy
Study on the Safety and Tolerability of STK-002 for Patients with Autosomal Dominant Optic Atrophy
This trial is investigating STK-002, an experimental medication designed to target the genetic cause of autosomal dominant optic atrophy. The condition affects the optic nerves, which carry visual information from the eyes to the brain, leading to progressive vision loss. STK-002 is a type of medicine called an antisense oligonucleotide, which works by binding to specific genetic material to modify how genes work in the body.
Who can participate: The study has two parts. Part A is for adults between 18 and 54 years old, while Part B is for younger patients between 6 and 17 years old. All participants must have a clinical diagnosis of ADOA and a confirmed genetic change in the OPA1 gene, which is responsible for maintaining healthy optic nerves. Participants must have a certain level of vision measured by the Best Corrected Visual Acuity score, which is the best vision someone can achieve with glasses or contact lenses. For most participants in Part A, this score needs to be between 35 and 70 letters on a standardized eye chart. However, the first two participants in the first group may have lower vision, with scores between 5 and 35 letters.
Who cannot participate: Patients with other eye diseases that could affect study results are excluded. Those who have had eye surgery within the past three months or who are taking medications that might interfere with the treatment cannot join. The trial also excludes patients with a history of severe allergic reactions to medications, pregnant or breastfeeding women, and those who have participated in another clinical trial within the last 30 days. Additionally, patients with serious health conditions that could make participation unsafe or those unable to follow study procedures are not eligible.
What the trial involves: The main goal is to evaluate whether STK-002 is safe and well-tolerated when given at different doses. The medicine is delivered through an injection directly into the eye. Researchers will monitor how the body processes the medication and track any potential side effects. Throughout the study, participants undergo various assessments to understand the treatment’s impact on vision and eye health. These include measuring the thickness of layers in the eye using a technique called optical coherence tomography, testing visual acuity (how clearly you can see), and evaluating contrast sensitivity (the ability to distinguish between different shades of light and dark). Participants will also complete questionnaires about their quality of life and vision-related experiences. The trial is expected to continue until the end of 2026.
Investigational drug: STK-002 is the experimental medication being tested. It is designed to help improve or stabilize vision by targeting the genetic cause of the condition at the molecular level. Researchers are studying different doses to determine the safest and most effective amount to use, as well as understanding how the medication is absorbed and processed in the body after being injected into the eye.
Summary
Currently, there is one active clinical trial for hereditary optic atrophy, focusing specifically on autosomal dominant optic atrophy caused by mutations in the OPA1 gene. This trial represents an important step in developing treatments for this genetic condition, which leads to progressive vision loss. The study is being conducted across multiple European countries, including Austria, Germany, Italy, and Denmark, providing opportunities for patients in these regions to access this experimental treatment. The trial tests STK-002, a genetically targeted therapy delivered through eye injections, and includes both adult and pediatric patients. Researchers are particularly interested in understanding the safety profile of this new treatment approach before moving to larger studies that would assess its effectiveness in preserving or improving vision.
