Randomized, Multicenter, Vehicle-Controlled, Double-Masked Study to Evaluate the Efficacy and Safety of Intranasal Cenegermin (Recombinant Human Nerve Growth Factor [rhNGF]) in Adult Participants with Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)
Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION) happens when blood flow to the optic nerve is suddenly reduced, leading to rapid sight loss. There are currently very few treatments available to help restore vision after this happens. This trial investigates whether an experimental nasal spray called cenegermin can help. Cenegermin is a lab-made version of a natural human protein that supports nerve survival and repair. Participants will be randomly chosen to receive either the real cenegermin spray or a dummy spray (placebo). Neither you nor your doctor will know which one you are receiving. Over 24 weeks, researchers will run regular eye checks, including reading standard eye charts and taking detailed scans of the back of your eye. The main goal is to find out if the treatment helps people read at least three extra lines (15 letters) on an eye chart.
At a glance
What is this study about?
Non-Arteritic Anterior Ischemic Optic Neuropathy, often shortened to NAION, is a condition that occurs when the blood supply to the optic nerve is suddenly reduced. The optic nerve acts like a cable carrying visual messages from your eye to your brain. When it loses its blood flow, nerve cells are starved of oxygen and become damaged, causing sudden and often permanent sight loss.
At present, there are very few proven medical options to help restore vision once NAION has occurred. This clinical trial is testing a medicine called cenegermin. Cenegermin is a synthetic version of a naturally occurring protein in the body known as nerve growth factor. In normal circumstances, this protein helps nerve cells grow, stay healthy, and heal from injury.
In this study, the medicine is given as a nasal spray so that it can travel close to the visual pathways. Researchers want to find out whether delivering this nerve-supporting protein can protect the optic nerve cells from further injury and encourage healing.
By comparing cenegermin directly against an inactive liquid spray over 24 weeks, doctors hope to gather clear evidence on whether this treatment improves vision and whether it can be used safely in patients with NAION in the future.
Key takeaways
- This Phase II study is testing a nasal spray called cenegermin for adults with NAION.
- Cenegermin is designed to mimic a natural body protein that helps protect and repair nerve cells.
- Half of the participants will receive the active medicine, while the other half will receive an inactive dummy spray.
- The main goal is to see if vision improves by at least 15 letters on an eye chart over 24 weeks.
- Participation requires regular eye tests and scans at a specialist hospital clinic.
- Joining the study is completely voluntary, and you can withdraw at any time.
Who may be eligible?
This study is open to adults aged 18 and over who have been diagnosed with NAION. Men and women of any background can take part if they meet the safety and medical criteria set by the research team.
To join, participants must have experienced vision loss from NAION and be well enough to attend regular clinic appointments. Because this study focuses on the health of the optic nerve, doctors will carefully check your eyes to make sure no other eye disease is causing your sight changes.
You may not be able to take part if you have certain other serious eye conditions, uncontrolled medical issues, or allergies to the study ingredients. The study doctor will review your complete medical history and perform detailed eye tests before confirming if the trial is right for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 years or older.
- I have been diagnosed by a doctor with NAION (Non-Arteritic Anterior Ischemic Optic Neuropathy).
- I am able and willing to use a daily nasal spray.
- I am able to attend regular clinic appointments for eye tests over at least 24 weeks.
- I do not have other major untreated eye diseases that severely affect my vision.
What does participation involve?
Taking part involves using a nasal spray as directed every day during the study. Because this is a controlled trial, you will be randomly assigned to receive either cenegermin or a harmless dummy spray with no active medicine. The trial lasts around 24 weeks, during which you will visit an eye clinic several times for close monitoring. At these visits, staff will test your vision using standard letter charts, check your side (peripheral) vision with light-detection tests, and take non-invasive photographic scans (known as OCT scans) to measure the thickness of the nerve layer in your eye. Staff will also check your general health, record any side effects you might experience, and answer any questions you have.
Potential risks and benefits
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Common questions
What is NAION?
NAION is a condition where a sudden drop in blood flow damages the optic nerve, causing sudden and painless loss of vision in one eye.
What is cenegermin?
Cenegermin is a lab-made copy of nerve growth factor, a natural protein in the body that helps nerve cells survive, heal, and stay healthy.
Will I definitely receive the real medicine?
No. This is a randomised study, meaning a computer randomly assigns you to receive either the active medicine or an inactive dummy spray.
Does the treatment involve injections into the eye?
No. The treatment being tested in this study is taken as a nasal spray, not an injection.
Can I leave the study if I change my mind?
Yes. You can withdraw from the study at any time, for any reason, without giving an explanation and without your normal medical care being affected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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