#1 Clinical Trials Search in Europe

18‐Mer Antisense Oligonucleotide Complementary To Opa1 Pre-Mrna

This article explores the ongoing clinical trial of STK-002, an innovative antisense oligonucleotide designed to treat Autosomal Dominant Optic Atrophy (ADOA). The study aims to evaluate the safety, tolerability, and effectiveness of this new medication in patients with this hereditary eye condition. We’ll delve into the trial’s objectives, eligibility criteria, and potential impact on patients’ lives.

Table of Contents

What is STK-002?

STK-002 is a new medication being developed to treat Autosomal Dominant Optic Atrophy (ADOA), a genetic eye disorder. It is classified as an 18‐MER antisense oligonucleotide that is designed to target a specific part of our genetic material called OPA1 pre-mRNA.[1]

The medication comes in the form of a solution for injection and is administered through intravitreal use, which means it is injected directly into the eye. This method allows the drug to reach the affected areas more effectively.[1]

What is Autosomal Dominant Optic Atrophy (ADOA)?

Autosomal Dominant Optic Atrophy (ADOA) is a genetic condition that affects the optic nerves, which carry visual information from the eyes to the brain. People with ADOA experience a gradual loss of vision over time. The condition is caused by changes (mutations) in a gene called OPA1, which is important for the health of cells in the optic nerve.[1]

How STK-002 Works

STK-002 is designed to work at the genetic level. It targets the OPA1 pre-mRNA, which is an intermediate form of genetic material that cells use to make the OPA1 protein. By interacting with this pre-mRNA, STK-002 aims to increase the production of healthy OPA1 protein, which could potentially slow down or prevent vision loss in people with ADOA.[1]

Clinical Trial Details

A clinical trial called “Osprey” is currently being conducted to study STK-002. This is a Phase I trial, which means it’s one of the earliest stages of testing a new drug in humans. The main goals of this study are to check if STK-002 is safe, how well patients tolerate it, and how the drug moves through the body after a single dose.[1]

Eligibility Criteria

To participate in this study, patients must meet certain criteria:

  • Age: 18 to 55 years old for Part A of the study, or 6 to 18 years old for Part B[1]
  • Confirmed diagnosis of ADOA with a specific type of genetic change in the OPA1 gene[1]
  • Certain levels of visual acuity (ability to see clearly)[1]

Some conditions that would prevent a person from participating include:

  • Having a different type of genetic change in the OPA1 gene[1]
  • Having symptoms of ADOA that affect parts of the body other than the eyes[1]
  • Having other eye conditions that could interfere with the study[1]
  • Being at risk for eye inflammation or infection[1]
  • Taking medications that could cause problems with the optic nerve[1]

Study Objectives

The main goals of this study are:

  1. To check if STK-002 is safe and well-tolerated when given as a single dose[1]
  2. To see how the drug moves through the body after being injected into the eye[1]
  3. To look for any changes in vision or eye structure after receiving STK-002[1]
  4. To see if STK-002 affects the quality of life for people with ADOA[1]

Safety and Effectiveness Measures

The researchers will be looking at several things to determine if STK-002 is safe and effective:

  • Safety: They will monitor for any side effects or problems that occur after the injection.[1]
  • Drug levels: They will measure how much STK-002 is in the blood at different times after the injection.[1]
  • Eye health: They will use special imaging techniques to look at the health of the retina and optic nerve.[1]
  • Vision tests: Participants will undergo various vision tests to check if there are any improvements in their eyesight.[1]
  • Quality of life: Participants will fill out questionnaires about how their vision affects their daily life.[1]

This clinical trial represents an important step in developing a potential treatment for ADOA, a condition for which there are currently no approved treatments. If successful, STK-002 could offer hope to people affected by this genetic eye disorder.

Aspect Details
Drug Name STK-002 (18‐Mer Antisense Oligonucleotide Complementary To Opa1 Pre-Mrna)
Condition Treated Autosomal Dominant Optic Atrophy (ADOA)
Trial Phase Phase I, First-In-Human study
Administration Method Intravitreal injection
Primary Objectives Evaluate safety, tolerability, and serum exposure of single doses
Secondary Objectives Assess changes in visual function, ocular structure, and quality of life
Eligibility Age Range Part A: 18-54 years, Part B: 6-17 years
Key Inclusion Criteria Clinical diagnosis of ADOA, confirmed OPA1 gene variant, specific visual acuity range
Key Exclusion Criteria Certain OPA1 variants, extraocular ADOA manifestations, specific ocular conditions
Outcome Measures Safety variables, pharmacokinetics, retinal thickness, visual acuity, contrast sensitivity, visual field, retinal electrical activity, reading acuity, quality of life

FAQ

  • What is Autosomal Dominant Optic Atrophy (ADOA)? ADOA is a hereditary eye condition that affects the optic nerve, leading to vision loss. It is caused by mutations in the OPA1 gene and is passed down through families in an autosomal dominant pattern.
  • What is STK-002 and how does it work? STK-002 is an 18-mer antisense oligonucleotide that targets the OPA1 pre-mRNA. It is designed to modify the expression of the OPA1 gene, which is responsible for ADOA. The medication is administered through intravitreal injection, meaning it's injected directly into the eye.
  • Who is eligible to participate in this clinical trial? The trial includes two parts: Part A for adults aged 18 to 54, and Part B for children and adolescents aged 6 to 17. Participants must have a clinical diagnosis of ADOA confirmed by genetic testing and meet specific visual acuity criteria.
  • What are the main objectives of this clinical trial? The primary objectives are to evaluate the safety and tolerability of single doses of STK-002 in ADOA patients and to determine its exposure in serum. Secondary objectives include assessing changes in visual function, ocular structure, and quality of life following treatment.
  • How will the effectiveness of STK-002 be measured? The trial will use various measurements to assess effectiveness, including changes in retinal nerve fiber layer thickness, visual acuity, contrast sensitivity, visual field, electrical activity of the retina, and reading acuity. Quality of life questionnaires will also be used to evaluate the treatment's impact on patients' daily lives.

Ongoing Clinical Trials on 18‐Mer Antisense Oligonucleotide Complementary To Opa1 Pre-Mrna

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

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Austria Denmark Germany Italy

Glossary

  • Autosomal Dominant Optic Atrophy (ADOA): A hereditary eye condition that affects the optic nerve, leading to vision loss. It is caused by mutations in the OPA1 gene and is inherited in an autosomal dominant pattern.
  • Antisense Oligonucleotide: A short piece of synthetic DNA or RNA that can bind to specific messenger RNA (mRNA) molecules and alter or inhibit gene expression.
  • Intravitreal Injection: A procedure where medication is injected directly into the vitreous cavity of the eye.
  • OPA1 Gene: A gene that provides instructions for making a protein involved in the function of mitochondria. Mutations in this gene are responsible for ADOA.
  • Best-Corrected Visual Acuity (BCVA): The best vision a person can achieve with the use of corrective lenses, measured using standardized eye charts.
  • Optical Coherence Tomography (OCT): A non-invasive imaging technique that uses light waves to take cross-section pictures of the retina.
  • Retinal Nerve Fiber Layer (RNFL): A layer of the retina containing nerve fibers that transmit visual information from the eye to the brain.
  • Ganglion Cell Layer (GCL): A layer of the retina containing nerve cells that process visual information before it's sent to the brain.
  • Contrast Sensitivity: The ability to distinguish between finer and finer increments of light versus dark.
  • Visual Field: The entire area that can be seen when the eye is focused on a central point.
  • Electroretinogram (ERG): A test that measures the electrical responses of various cell types in the retina.
  • Phototopic Negative Response (PhNR): A component of the electroretinogram that reflects the activity of retinal ganglion cells.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-stk-002-for-patients-with-autosomal-dominant-optic-atrophy/