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A Study of Aganirsen Eye Drops for Patients with Corneal Blood Vessel Growth in Aniridia-Associated Keratopathy

Verified siteRegistered drugNo placebo
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What is this trial about?

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

This study examines a condition called Aniridia-Associated Keratopathy, which is a disease affecting the front clear part of the eye known as the cornea. In this condition, abnormal blood vessels grow into the cornea, which is normally clear and free of blood vessels. This abnormal growth of blood vessels is called Corneal Neovascularization and it occurs in people who have aniridia, a rare condition where part or all of the colored part of the eye is missing from birth. The treatment being tested is Olisens, which is also known by its scientific name aganirsen or code name GS101. This medication is an eye drop solution that works by blocking a specific substance in the body called IRS-1, which is involved in the formation of new blood vessels.

The purpose of this study is to test whether Olisens eye drops can stop or reduce the abnormal blood vessel growth in the cornea of people with Aniridia-Associated Keratopathy. The main goal is to measure if the treatment can decrease the area of the cornea that is covered by these unwanted blood vessels. During the study, participants will receive the eye drop treatment for up to 12 weeks, with a maximum daily dose of 0.52 milligrams. The study will track changes in the blood vessel coverage on the cornea using special photographs taken with a device called a slit lamp, which allows doctors to examine the eye in detail.

Throughout the study, several other measurements will be taken to understand how the treatment affects the eyes and the quality of life of participants. These include checking how well participants can see using special eye charts, measuring the pressure inside the eye, examining the surface of the eye for any damage, testing how sensitive the cornea is to touch, and measuring the thickness of the cornea. The study will also look at the characteristics of the blood vessels themselves, such as their thickness and length, and whether they shrink or come back after treatment. Additionally, participants will complete questionnaires about their vision-related quality of life and any eye pain they experience. The back part of the eye called the retina will also be examined to check for any unwanted effects of the treatment.

The research process

The trial runs in 11 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Treatment period with eye drops

    During the trial, Olisens eye drops will be administered to your affected eye. The eye drops contain aganirsen at a concentration of 0.86 mg/ml.

    The eye drops are provided in single-dose containers, which means each container is used once and then discarded.

    The eye drops are designed to target and reduce the abnormal blood vessels growing on the cornea, which is the clear front surface of your eye.

    The specific dosage schedule, including how many times per day the drops should be applied and for how long, will be determined according to the trial protocol.

    Only one eye will receive treatment, even if both eyes are affected by the condition.

  2. Step 2

    Regular monitoring visits

    Throughout the trial, regular visits will be scheduled to monitor the progress and safety of the treatment.

    During these visits, various measurements and examinations will be performed to assess how the treatment is working and to check for any side effects.

  3. Step 3

    Corneal blood vessel assessment

    The main focus of the trial is to measure changes in the abnormal blood vessels on the cornea.

    Photographs of the front of your eye will be taken using a specialized microscope called a slit lamp.

    These images will be analyzed to determine the percentage of the cornea covered by blood vessels and whether this area is decreasing with treatment.

    Additional measurements will be taken to assess vessel thickness, vessel length, whether vessels are shrinking, and whether any new vessels are appearing.

  4. Step 4

    Vision testing

    Your visual acuity, which is the sharpness of your vision, will be tested using standardized eye charts called ETDRS charts.

    This includes testing your vision under normal conditions and also with glare, which means testing how well you see when there is bright light that might cause discomfort.

    The results will be recorded using a measurement system called logMAR.

  5. Step 5

    Eye pressure measurement

    The pressure inside your eye, known as intraocular pressure, will be measured.

    This will be done using a device called I Care or Goldmann applanation tonometry.

    The pressure will be recorded in units called mmHg.

  6. Step 6

    Corneal surface examination

    The surface of your cornea will be examined for any damage or staining.

    This assessment uses the Oxford Grading Scale, which rates the condition on a scale from 0 to 5.

    The stage of your condition will also be assessed using the Lagali Aniridia Associated Keratopathy Grading system.

  7. Step 7

    Corneal sensitivity testing

    The sensitivity of your cornea will be tested using a device called a Cochet-Bonnet esthesiometer.

    This device uses a thin nylon thread that gently touches the cornea to measure how well you can feel touch on the surface of your eye.

  8. Step 8

    Corneal thickness measurement

    The thickness of the central part of your cornea will be measured.

    This will be done using specialized imaging equipment called Scheimpflug Corneal Tomography (Pentacam) or Anterior Segment Optical Coherence Tomography (Casia-2).

  9. Step 9

    Retinal safety check

    The retina, which is the light-sensitive layer at the back of your eye, will be examined to check for any adverse effects.

    This will be done using Optical Coherence Tomography, an imaging technique that creates detailed pictures of the retina.

  10. Step 10

    Infection and ulcer assessment

    Your eye will be examined using a slit lamp to check for any signs of active infectious keratitis, which is an infection of the cornea, or corneal ulceration, which is an open sore on the cornea.

  11. Step 11

    Quality of life questionnaires

    You will be asked to complete questionnaires to assess how your vision and eye condition affect your daily life.

    These include the NEI-VFQ25, which evaluates vision-related quality of life.

    You will also complete the Aniridia-Net questionnaire, which is specific to people with aniridia.

    Additionally, the Ocular Pain Assessment Survey (OPAS) will be used to evaluate any eye pain you may be experiencing.

Who can join the trial?

8 criteria

  • You must have corneal neovascularization, which means abnormal blood vessels growing into the clear front part of your eye, caused by a condition called aniridia-associated keratopathy, a disease affecting the cornea in people born without a complete iris.
  • You must be at least 12 years old or older, and can be male or female.
  • You or your parents or legal guardian must sign a written informed consent form, which is a document that shows you understand and agree to join the study, before any study procedures begin.
  • You must have a confirmed diagnosis of aniridia, which is a condition where part or all of the colored part of the eye is missing, and you must have progressive corneal neovascularization, meaning the abnormal blood vessels in your cornea are getting worse over time. This must be confirmed by either two photographs taken at least four weeks apart or by your doctor's examination. Alternatively, you may have Stage 2 aniridia-associated keratopathy, which is a specific level of corneal disease.
  • You must have at least a 2 millimeter diameter area in the center of your cornea that is clear and does not have blood vessels growing into it.
  • You must not have any other medical reasons that would prevent you from receiving any part of the treatment being studied.
  • You must be willing and able to cooperate and follow the study procedures and attend all required follow-up visits.
  • If both of your eyes have the disease, only one eye will be included in the study.

Who cannot join the trial?

3 criteria

  • The study information does not list specific reasons why patients cannot participate in this clinical trial
  • If you are interested in participating, the research team will need to review your individual medical situation to determine if you are eligible
  • General factors that might prevent participation could include other eye conditions, certain medications you are taking, or other health problems, but these specific details are not provided in the available study information
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Investigated drugs

Olisens is an eye drop medication that contains an antisense oligonucleotide designed to work against a specific target called IRS-1. This medication is being tested to see if it can stop or reduce the growth of abnormal blood vessels in the cornea (the clear front part of the eye) in patients with a condition called aniridia-associated keratopathy. The eye drops are applied directly to the eye to help treat this blood vessel problem.

What is already known about the treatment

Olisens – Olisens is an experimental eye drop medication containing an antisense oligonucleotide designed to target a specific protein called IRS-1, which is currently being studied in clinical trials for treating abnormal blood vessel growth in the cornea of patients with a rare eye condition called aniridia-associated keratopathy. The medication works at the molecular level by blocking the production of IRS-1 protein, which plays a role in the development of unwanted blood vessels that can cloud the normally clear cornea and impair vision. It is administered as eye drops directly onto the surface of the eye, and the main goal of treatment is to stop or reduce the growth of these abnormal blood vessels in the cornea. This medication belongs to the class of antisense oligonucleotide therapies, which are designed to interfere with specific genetic instructions in cells to prevent the production of disease-causing proteins.

Investigated diseases

Corneal Neovascularization in Aniridia-associated Keratopathy – This condition involves the abnormal growth of blood vessels into the cornea, which is the clear front surface of the eye, occurring in patients who have aniridia-associated keratopathy. Aniridia is a rare genetic disorder characterized by the partial or complete absence of the iris, the colored part of the eye. People with aniridia often develop keratopathy, which means disease or damage to the cornea. As the keratopathy progresses, new blood vessels begin to grow from the edge of the cornea toward its center, a process called neovascularization. These vessels cloud the normally clear cornea and interfere with vision. The condition gradually worsens over time, with the vascularized area of the cornea potentially expanding and affecting larger portions of the corneal surface.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-522609-39-00Protocol codeUni-Koeln-5378Estimated enrolment20 patientsSponsorUniversity Of Cologne

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