In short
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.
At a glance
- 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
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.
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.
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.
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.
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.
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
- Confirmed diagnosis of ADOA with a specific type of genetic change in the OPA1 gene
- Certain levels of visual acuity (ability to see clearly)
Some conditions that would prevent a person from participating include:
- Having a different type of genetic change in the OPA1 gene
- Having symptoms of ADOA that affect parts of the body other than the eyes
- Having other eye conditions that could interfere with the study
- Being at risk for eye inflammation or infection
- Taking medications that could cause problems with the optic nerve
Study Objectives
The main goals of this study are:
- To check if STK-002 is safe and well-tolerated when given as a single dose
- To see how the drug moves through the body after being injected into the eye
- To look for any changes in vision or eye structure after receiving STK-002
- To see if STK-002 affects the quality of life for people with ADOA
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.
- Drug levels: They will measure how much STK-002 is in the blood at different times after the injection.
- Eye health: They will use special imaging techniques to look at the health of the retina and optic nerve.
- Vision tests: Participants will undergo various vision tests to check if there are any improvements in their eyesight.
- Quality of life: Participants will fill out questionnaires about how their vision affects their daily life.
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.