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Efficacy and Safety of Intranasal Cenegermin in Adults with Non‑Arteritic Anterior Ischemic Optic Neuropathy (NAION)

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What is this trial about?

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

Non-Arteritic Anterior Ischemic Optic Neuropathy is a rare eye condition that causes a sudden loss of vision because the optic nerve does not receive enough blood flow. The study looks at an intranasal spray called cenegermin, which is a laboratory‑made version of a protein that helps nerve cells grow. Participants will be given either the active spray or a similar‑appearing inactive spray (placebo) that does not contain the drug.

The purpose of the study is to find out whether the spray can improve the sharpness of vision, known as visual acuity, compared with the placebo. Over several months, participants will use the spray daily and attend regular visits where standard eye tests are performed, such as reading letters on a chart, checking the area that can be seen without moving the eyes (visual field), and an eye scan called optical coherence tomography that measures the thickness of retinal layers. The study follows each person from the start of treatment through the end of the observation period to see how vision changes over time.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial enrollment

    After you agree to take part, you receive a study identification number and a copy of the study information.

    The study staff explains the purpose of the trial, what will be required of you, and answers any questions you have.

  2. Step 2

    Random assignment

    You are randomly placed into one of two groups without knowing which group you are in.

    One group receives cenegermin (the active medication) and the other group receives a matching vehicle (placebo).

  3. Step 3

    Baseline assessments

    Before any medication is started, you undergo a series of eye tests.

    These include a measurement of best corrected visual acuity using a standardized chart, visual‑field testing, and imaging of the eye layers with optical coherence tomography.

  4. Step 4

    Start of intranasal treatment

    You begin using a nasal spray that contains either cenegermin or the vehicle.

    The spray is administered through the nose according to the schedule defined by the study protocol and will be continued for a total of 24 weeks.

    The exact dose amount and frequency are specified in the study instructions that you receive.

  5. Step 5

    Regular follow‑up visits

    Throughout the 24‑week treatment period you attend scheduled clinic visits.

    At each visit the study team checks your overall health, records any side effects, and repeats the visual acuity and visual‑field tests to monitor progress.

  6. Step 6

    Final evaluation at week 24

    At the end of the 24‑week period you undergo the same set of eye examinations performed at baseline.

    The results are used to determine whether there has been an improvement of at least 15 letters on the visual‑acuity chart and other secondary outcomes.

  7. Step 7

    Study completion

    After the final assessment, your participation in the trial ends.

    The study staff provides you with a summary of the overall findings and any further recommendations for your eye care.

Who can join the trial?

7 criteria

  • Age: You must be between 50 and 80 years old when you sign the consent form.
  • Diagnosis of NAION: You need a confirmed diagnosis of non‑arteritic anterior ischemic optic neuropathy affecting only one eye, with symptoms starting before the first dose. At the screening visit this must be shown by all of the following: swelling of the optic nerve head (ONH edema); a visual field test pattern that matches NAION, reviewed by a central reading center; an average visual field sensitivity between 7 and 30 decibels (a decibel measures how well you can detect light spots); an eye scan called optical coherence tomography (OCT) that shows typical changes; and an unequal pupil response known as a relative afferent pupillary defect in the study eye (this last test is not required if the other eye already had NAION or another eye disease that is allowed).
  • Timing of first dose: You must be able to receive the first dose of the study drug within 14 days after your symptoms began.
  • Vision test score: Your best corrected visual acuity (BCVA) in the study eye must be between 15 and 65 letters when measured with an ETDRS chart (a standard eye‑letter chart).
  • Clear eye media and pupil dilation: The clear parts of your eye (the “ocular media”) must allow the doctor to see the optic nerve and retina, and your pupils must be able to dilate enough for a proper exam.
  • Informed consent: You must be able to understand the study information and sign a consent form agreeing to follow the study rules.
  • Eligibility of men and women: Both men and women can join, but women must not be pregnant or breastfeeding. If you are a woman who could become pregnant (childbearing potential), you must use an acceptable method of birth control during the study and for at least 30 days after the last dose. You also need a negative pregnancy test (urine or blood) at screening and again before the first dose; if a urine test is unclear, a blood test will be done, and a positive result means you cannot take part.

Who cannot join the trial?

19 criteria

  • Having NAION in both eyes, or the other eye becoming affected within 6 weeks of the study eye, means you cannot join the study.
  • Signs of temporal arteritis (also called giant cell arteritis) such as jaw pain when chewing, tenderness of the scalp or temples, swelling of the optic nerve head, or a combination of symptoms like transient vision loss, headache, muscle aches, loss of appetite, weight loss, or fever exclude you.
  • Lab tests that suggest temporal arteritis, including a C‑reactive protein level more than twice the normal limit, an elevated erythrocyte sedimentation rate (a blood test that rises with inflammation), or a platelet count over 450,000 per microliter, also make you ineligible.
  • Experiencing pain when moving your eye is an exclusion criterion.
  • If your blood hemoglobin level is below 10 g/dL (a measure of how many red blood cells you have), you cannot participate.
  • Having an intraocular pressure higher than 25 mm Hg (the pressure inside the eye) or a history of glaucoma in the study eye excludes you.
  • Having intermediate age‑related macular degeneration (AMD) with certain types of deposits, the wet form of AMD, or advanced AMD (geographic atrophy) in the study eye disqualifies you.
  • Uncontrolled diabetes (hemoglobin A1c ≥ 8.0 %), any level of diabetic eye disease, or prior laser treatment to the retina or macula makes you ineligible.
  • Any history of optic neuritis (inflammation of the optic nerve), any type of intraocular inflammation (such as uveitis), infections, nutritional or hereditary optic nerve damage, radiation‑related eye damage, tumors involving the eye, active eye infection, multiple sclerosis, collagen‑vascular disease, other chronic inflammatory diseases, or chronic immunosuppressive conditions excludes you.
  • Having amblyopia (often called “lazy eye”) or any other reason for vision loss in the study eye that is not related to NAION prevents participation.
  • Any current or past cancer, including known or suspected eye tumors, systemic cancers that could affect your ability to follow the study, a history of cancer treated within the past 5 years (except a non‑facial basal cell skin cancer that has been fully treated), or any history of cancer of the brain or spinal cord excludes you.
  • Use of certain medicines before you sign the consent form also disqualifies you, such as systemic corticosteroids (steroids taken by mouth or injection) within 1 month, intranasal drugs used for more than 7 days in the past month (except saline sprays), neuroprotective agents (like nicotinamide, memantine, apraclonidine, or brimonidine) within 6 weeks, immunomodulatory therapy (drugs that change the immune system) within 3 months, amiodarone (a heart medication) within 12 months, chemotherapy within 3 years, head or neck radiation therapy within 20 years, or medications known to harm the optic nerve or retina (such as ethambutol, hydroxychloroquine, or chloroquine).
  • Planning to use phosphodiesterase‑5 (PDE‑5) inhibitors (medications such as Viagra) during the study period makes you ineligible.
  • Conditions that would stop the nasal spray from being delivered properly, like a history of nasal polyps, chronic sinusitis, structural blockage of the nasal passages, or previous nasal or sinus surgery, exclude you.
  • Active substance use disorder or dependence on alcohol, illegal drugs, misuse of prescription medications, marijuana, or cannabis‑derived products disqualifies you.
  • If a reliable best‑corrected visual acuity (BCVA) measurement cannot be obtained in the study eye—meaning two measurements are not within five letters of each other—you cannot take part.
  • Being enrolled in another clinical trial within 3 months before signing consent, or planning to join another trial during this study, excludes you.
  • Having a known allergic reaction or significant hypersensitivity to any drug or ingredient used in this study prevents participation.
  • Any other eye disease, systemic disease, active psychiatric illness, or condition that the investigator believes could affect how the treatment works, interfere with interpreting the results, or make it difficult to attend study visits or complete study procedures makes you ineligible.
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Investigated drugs

Cenegermin is a medicine made from a lab‑produced version of human nerve growth factor. In this study it is given as a nasal spray that turns into a solution inside the nose. The goal of using cenegermin is to see if it can help the nerves in the eye recover after a type of optic nerve damage called non‑arteritic anterior ischemic optic neuropathy (NAION). Researchers are testing whether people who receive cenegermin have better eyesight compared with those who receive only the spray’s inactive ingredients. The medication is being examined for both how well it works and how safe it is when used in this way.

What is already known about the treatment

Cenegermin - It is given as a nasal spray that is prepared from a powder and sprayed into the nose. The drug is an approved treatment for severe eye surface disease and is also being studied in clinical research for optic nerve damage, so it is a known medicine with published studies. It is used mainly to help heal damaged nerve cells in the eye and may improve vision loss in NAION. Cenegermin works by acting like the body’s natural nerve growth factor, binding to nerve receptors and encouraging nerve cell survival and repair; it belongs to the class of recombinant protein growth factors.

Investigated diseases

Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION) - NAION is a sudden loss of vision caused by reduced blood flow to the front part of the optic nerve. It usually affects one eye first and can involve the other eye later. Vision loss often appears upon waking and may include blurry vision and reduced color perception. Swelling of the optic nerve can lead to gradual thinning of nerve fibers over weeks to months. Visual field defects may expand as the damage progresses.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-524748-37-00Protocol codeNGF-NAION-301Estimated enrolment408 patientsSponsorDompe' Farmaceutici S.p.A.

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