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Study on the Safety and Tolerability of STK-002 for Patients with Autosomal Dominant Optic Atrophy

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Autosomal Dominant Optic Atrophy (ADOA). ADOA is a genetic eye disease that affects the optic nerves, which are responsible for transmitting visual information from the eyes to the brain. The study will investigate a new treatment called STK-002, which is a type of medicine known as an antisense oligonucleotide. This treatment is designed to target specific genetic material related to the disease.

The purpose of the study is to evaluate the safety and tolerability of different doses of STK-002 in patients with ADOA. The study will also look at how the body processes the medicine after it is given as an injection into the eye, a method known as intravitreal use. Participants will receive single doses of the study medicine, and researchers will monitor them to gather information about the treatment's effects and any potential side effects.

Throughout the study, various assessments will be conducted to understand the impact of the treatment on vision and eye health. These assessments include measuring the thickness of certain layers in the eye using a technique called optical coherence tomography (OCT), evaluating visual acuity, which is the clarity or sharpness of vision, and testing contrast sensitivity, which is the ability to distinguish between different shades of light and dark. Additionally, the study will assess the quality of life of participants using specific questionnaires. The trial is expected to continue until the end of 2026.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age and a clinical diagnosis of autosomal dominant optic atrophy (ADOA).

    A genetic test is performed to confirm a specific gene variant related to ADOA.

  2. Step 2

    Baseline measurements

    Baseline measurements are taken to assess eye health. These include tests for visual acuity, contrast sensitivity, and visual field.

    Additional tests may include measuring the thickness of certain layers in the eye and assessing electrical activity in the retina.

  3. Step 3

    Administration of STK-002

    The study medicine, STK-002, is administered as a single dose through an injection into the eye (intravitreal use).

    The dosage may vary as the study investigates different dose levels.

  4. Step 4

    Monitoring and follow-up

    After administration, monitoring occurs to evaluate safety and how the body processes the medicine.

    Follow-up visits include repeating some of the initial tests to monitor changes in vision and eye health.

  5. Step 5

    Completion of study

    The study concludes with a final assessment to gather data on the safety and effects of the medicine.

    Participants may be asked to complete questionnaires about their quality of life and vision-related experiences.

Who can join the trial?

3 criteria

  • The patient must be between **18 and 54 years old** to participate in Part A, and between **6 and 17 years old** to participate in Part B.
  • The patient must have a **clinical diagnosis** of Autosomal Dominant Optic Atrophy (ADOA) and a specific change in the **OPA1 gene** confirmed by a lab test during screening.
  • The patient must have a **Best Corrected Visual Acuity (BCVA)** score between **35 and 70** letters in each eye, except for the first two patients in Cohort 1 of Part A, who must have a BCVA score between **5 and 35** letters in each eye. BCVA is a measure of the best vision a person can achieve with glasses or contact lenses.

Who cannot join the trial?

8 criteria

  • Patients who have any other eye diseases that could affect the study results.
  • Patients who have had eye surgery in the past three months.
  • Patients who are currently using any medication that might interfere with the study treatment.
  • Patients with a history of severe allergic reactions to medications.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial within the last 30 days.
  • Patients with any serious health conditions that could make participation unsafe.
  • Patients who are unable to follow the study procedures or attend all required visits.
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Investigated drugs

STK-002 is an experimental medication being studied for its potential to treat Autosomal Dominant Optic Atrophy (ADOA). It is an antisense oligonucleotide, which means it is designed to bind to specific genetic material in the body. The goal of this medication is to help improve or stabilize vision in patients with ADOA by targeting the underlying genetic cause of the condition. In this trial, researchers are investigating the safety and tolerability of different doses of STK-002 when administered directly into the eye. They are also studying how the medication is absorbed and processed in the body after administration.

What is already known about the treatment

STK-002 – This medication is administered through an injection directly into the eye, known as intravitreal injection. It is currently being studied in clinical trials to assess its safety and how well it is tolerated in patients with Autosomal Dominant Optic Atrophy (ADOA). The main therapeutic use being explored is for the treatment of ADOA, a genetic condition affecting vision. STK-002 works at the molecular level as an antisense oligonucleotide, which means it is designed to bind to specific RNA molecules to modify gene expression. It falls under the pharmacological classification of genetic therapies, specifically targeting genetic disorders.

Investigated diseases

Autosomal Dominant Optic Atrophy – This is a genetic condition that primarily affects the eyes, leading to progressive vision loss. It is caused by mutations in the OPA1 gene, which is responsible for maintaining the health of the optic nerves. Individuals with this condition typically experience a gradual decrease in visual acuity, often starting in childhood or early adulthood. The disease may also cause color vision defects and a reduction in the field of vision. Over time, the optic nerve fibers degenerate, leading to further vision impairment. The severity of vision loss can vary widely among affected individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2023-506290-35-00Protocol codeSTK-002-OA-101Estimated enrolment58 patientsSponsorStoke Therapeutics Inc.

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