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

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What is this study about?

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.

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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medical University Of Vienna Vienna Austria

Other Sites

Site Name City Country Status
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Ospedale San Raffaele S.r.l. Milan Italy
Justus-Liebig-Universitaet Giessen Giessen Germany
Rigshospitalet Copenhagen Denmark

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Recruiting
15.03.2024
Denmark Denmark
Recruiting
15.03.2024
Germany Germany
Recruiting
15.03.2024
Italy Italy
Not yet recruiting
15.03.2024

Trial locations

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.

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 ID:
2023-506290-35-00
Protocol code:
STK-002-OA-101
Trial Phase:
Human Pharmacology (Phase I) – First administration to humans

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