Non-Arteritic Anterior Ischemic Optic NeuropathyPhase 3NCT07453888
Efficacy and Safety of Intranasal Cenegermin in Adult Participants With Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)
A Phase 3 study testing Cenegermin in people with Non-Arteritic Anterior Ischemic Optic Neuropathy, run by Dompé Farmaceutici S.p.A at 38 sites in 4 countries.
Trial summary
This is a phase 3, randomized, multicenter, vehicle-controlled, double-masked study to evaluate the efficacy and safety of intranasal cenegermin compared with vehicle control in adult participants with NAION. Read more about this study
- Who is it for?
- Ages 50 to 80 years
- Conditions the study is for
- Non-Arteritic Anterior Ischemic Optic Neuropathy
- What is being tested?
- Cenegermin
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 272 participants
- Where
- 38 sitesin 4 countries
- Study dates
- 1 Jul 2026to Sep 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A clinical diagnosis of unilateral NAION in the study eye with symptom onset within 14 days prior to the planned date for first dose administration.
- A BCVA score in the study eye of ≥ 15 letters and ≤ 65 letters measured using the ETDRS chart.
- Sufficiently clear ocular media and adequate pupil dilation to enable assessment of the optic nerve and retina in both eyes.
Who cannot join
- Bilateral NAION or sequential NAION with fellow eye involvement within 6 weeks of study eye involvement.
- Clinical evidence of temporal arteritis (giant cell arteritis) signs or symptoms.
- Abnormal laboratory findings suggestive of temporal arteritis (giant cell arteritis), in the absence of a known acute cause
- Pain with eye movement
- Intraocular pressure (IOP) greater than 25 mmHg in the study eye or history of glaucoma in the study eye.
- Intermediate age-related macular degeneration (AMD) with subfoveal drusen, exudative AMD, or geographic atrophy in the study eye.
- Note: Additional inclusion/exclusion criteria apply, as defined in the protocol.
Trial locations and contacts
38 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
Dompé farmaceutici S.p.A. Via Santa Lucia, 6, 20122 Milan (MI) contact
38 sites in 4 countries
United States27 sites, 27 recruiting
- Recruiting
University of Michigan Health - Kellogg Eye Center
Ann Arbor, Michigan, 48105
734-763-0482, ldelott@med.umich.edu
Lindsey DeLott
- Recruiting
Emory University - Emory Eye Center - Atlanta
Atlanta, Georgia, 30322
404-778-2020, michael.dattilo@emory.edu
Michael Dattilo
- Recruiting
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
303-724-9008, prem.subramanian@ucdenver.edu
Prem Subramanian
- Recruiting
The Johns Hopkins Hospital (JHH)
Baltimore, Maryland, 21287
667-306-7723, ahende24@jhmi.edu
Amanda Henderson
- Recruiting
Tufts Medical Center - Neurology
Boston, Massachusetts, 02116
617-636-5489, Laurel.Vuong@tuftsmedicine.org
Laurel Vuong
- Recruiting
Retina Consultants of South Carolina, Charleston Neuroscience Institute- West Ashley (RCA)
Charleston, South Carolina, 29414
843-763-4466, john.kerrison@retinasouthcarolina.com
John Kerrison
- Recruiting
Northwestern University - Ophthalmology
Chicago, Illinois, 60611
312-926-0747, shira.simon@northwestern.edu
Shira Simon
- Recruiting
University of Illinois at Chicago Eye and Ear Infirmary - Ophthalmology
Chicago, Illinois, 60612
312-413-7491, Pmacint1@uic.edu
Peter MacIntosh
- Recruiting
Duke Eye Center - Ophthalmology
Durham, North Carolina, 27705
919-668-0558, sid.gospe@duke.edu
Sidney Gospe
- Recruiting
Trinity Health Grand Rapids
Grand Rapids, Michigan, 49503
616-685-6306, santosro@trinity-health.org
Ronel Santos
- Recruiting
Neuro Eye Clinical Trials
Houston, Texas, 77074
346-429-4135, rosatang.md@me.com
Rosa Tang
- Not yet recruiting
Univ. Of Kentucky Clinic Dept of Ophthalmology & Vision Science
Lexington, Kentucky, 40508
859-323-0028, padmaja.sudhakar@uky.edu
Padmaja Sudhakar
- Recruiting
Neuro Eye Orbit Institute
Los Angeles, California, 90048
310-469-9080, sbose@neuroeyeorbit.com
Swaraj Bose
- Not yet recruiting
David Geffen School of Medicine at UCLA - Ophthalmology
Los Angeles, California, 90095
626-817-4701, quiros@jsei.ucla.edu
Peter Quiros
- Not yet recruiting
Bascom Palmer Eye Institute, University of Miami School of Medicine
Miami, Florida, 33136
305-243-1250, blam@med.miami.edu
Byron Lam
- Recruiting
University of Minnesota, Dept of Ophthalmology
Minneapolis, Minnesota, 55455
612-273-6212, mikelee@umn.edu
Michael Lee
- Recruiting
California Orbital Consultants
Newport Beach, California, 92660
626-475-0989, Madhu.agarwal@nvisioncenters.com
Madhu Agarwal
- Recruiting
UNMC Truhlsen Eye Institute
Omaha, Nebraska, 68105
402-559-1852, slynch@unmc.edu
Shannon Lynch
- Recruiting
Stanford Byers Eye Institute
Palo Alto, California, 94303
650-723-6995, yjliao@stanford.edu
Yaping Joyce Liao
- Recruiting
University of Pennsylvania - Ophthalmology
Philadelphia, Pennsylvania, 19104
215-662-8696, madhura.tamhankar@pennmedicine.upenn.edu
Madhura Tamhankar
- Not yet recruiting
Wills Eye Hospital
Philadelphia, Pennsylvania, 19107
215-825-4787, Danijel.j.pericic@gmail.com
Danijel Pericic
- Recruiting
Mayo Clinic - Department of Ophthalmology
Rochester, Minnesota, 55905
480-574-1160, chen.john@mayo.edu
John Chen
- Recruiting
UC Davis Health, Ophthalmology
Sacramento, California, 95817
800-482-3284, aycliu@health.ucdavis.edu
Yin Allison Liu
- Recruiting
Sarasota Retina Institute - Research
Sarasota, Florida, 34239
941-921-5335, sri30research@aol.com
Marc Levy
- Recruiting
Washington University School of Medicine in St Louis - Ophthalmology
St Louis, Missouri, 63108
314-286-2946, vanstaverng@wustl.edu
Gregory Van Stavern
- Recruiting
University of South Florida/Ophthalmology
Tampa, Florida, 33612
813-974-2020, asriram2@usf.edu
Aishwarya Sriram
- Recruiting
Retina Consultants of Texas - The Woodlands
The Woodlands, Texas, 77384
713-524-3434, svsmd@retinaconsultantstexas.com
Stacy Smith
United Kingdom6 sites, 6 recruiting
- Recruiting
Oxford University Hospital NHS Trust - John Radcliffe Hospital
Abingdon, Oxfordshire, OX3 9DU
4400000000, srilakshmi.sharma@ouh.nhs.uk
Srilakshmi Sharma
- Recruiting
Bristol NHS Foundation Trust - Bristol Eye Hospital
Bristol, BS1 2LX
01173424770, simon.epps@uhbw.nhs.uk
Simon Epps
- Recruiting
Liverpool University Hospitals NHS Foundation Trust - Royal Liverpool University Hospital - St Paul's Eye Unit
Liverpool, L7 8TX
0151 706 3874, nima.ghadiri@liverpoolft.nhs.uk
Nima Ghadiri
- Recruiting
Western Eye Hospital
London, Greater London, NW1 5QH
4401753636210, richard.nicholas3@nhs.net
Richard Nicholas
- Recruiting
Manchester University NHS Foundation Trust - Manchester Royal Eye Hospital
Manchester, Greater Manchester, M13 9WL
01617010826, ali.yagan@mft.nhs.uk
Ali Yagan
- Recruiting
Sheffield Teaching Hospitals NHS Foundation Trust - Royal Hallamshire Hospital
York, Sheffield, S10 2JF
4401142712619, simon.hickman@nhs.net
Simon Hickman
South Korea3 sites, 3 recruiting
- Recruiting
Pusan National University Hospital - Ophthalmology
Busan, Busan Gwang'yeogsi, 49241
82-51-240-7000, hychoi@pusan.ac.kr
Hee-young Choi
- Recruiting
Asan Medical Center - Ophthalmology
Seoul, Seoul Teugbyeolsi, 05505
82-2-3010-5001, bjlee99@amc.seoul.kr
Byung Joo Lee
- Recruiting
Samsung Medical Center - Ophthalmology
Seoul, Seoul Teugbyeolsi, 06351
82-2-3410-0200, kyungahh.park@samsung.com
Kyung Ah Park
Australia2 sites, 2 recruiting
- Recruiting
Cerulea
East Melbourne, Victoria, 3002
0399590051, shivanand.sheth@eyeandear.org.au
Shivanand Sheth
- Recruiting
Vision SA - Ophthalmology
Kent Town, South Australia, 5067
61882044899, celia.chen@health.sa.gov.au
Celia Chen
About this study
Official title: Randomized, Multicenter, Vehicle-Controlled, Double-Masked Phase 3 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)
This is a phase 3, randomized, multicenter, vehicle-controlled, double-masked study to evaluate the efficacy and safety of intranasal cenegermin compared with vehicle control in adult participants with NAION. Approximately 272 participants who meet all eligibility criteria will be randomly assigned in a 1:1 ratio to receive either cenegermin treatment (Group 1) or the vehicle control (Group 2).
- Being tested
- Cenegermin, Vehicle
- Keywords
- Acute optic neuropathy, Non-Arteritic Anterior Ischemic Optic Neuropathy, NAION, cenegermin, Optic neuropathy, Intranasal
- Medical subject headings
- Optic Neuropathy, Ischemic, Optic Nerve Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CenegerminDrug
Cenegermin is administered intranasally.
VehicleOther
Vehicle spray is administered intranasally.
| Study group | Role | What they receive |
|---|---|---|
| Cenegermin | Experimental | Participants will be administered with cenegermin spray in each nostril. Receives: Drug: Cenegermin |
| Vehicle | Placebo comparator | Participants will be administered with vehicle spray in each nostril. Receives: Other: Vehicle |
What is being measured
The primary outcome is the main question the study is designed to answer.
Achievement of ≥ 15 letter increase in Best Corrected Visual Acuity (BCVA), assessed in each individual participant, measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
Improvement in visual acuity defined as a >=15 letter increase in BCVA using ETDRS chart.
Measured: Baseline to Week 24
Secondary outcomes (5)
Change from baseline in visual field mean sensitivity in decibels
Change from baseline in visual field mean sensitivity in decibels will be analyzed
Measured: Baseline through Week 24
Change from baseline in BCVA as measured by the ETDRS chart
Measured: Baseline through Week 24
Change from baseline in visual field mean sensitivity, in a prespecified region consisting of at least 5 separate loci on visual field testing
Change from baseline in mean sensitivity at week 24 in loci prespecified at baseline will be analyzed.
Measured: Baseline through Week 24
Change from baseline in ganglion cell layer-inner plexiform layer (GCL-IPL) thickness (micrometer [μm]) on optical coherence tomography (OCT).
Change from baseline in GCL-IPL thickness on optical coherence tomography will be analyzed.
Measured: Baseline through Week 24
Incidence of ocular and non-ocular adverse events
Number of participants with ocular and non-ocular adverse events will be assessed.
Measured: Through Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2026
- Study start1 Jul 2026
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Non-Arteritic Anterior Ischemic Optic NeuropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 272 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dompé Farmaceutici S.p.A
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07453888
- Sponsor's protocol ID
- NGF-NAION-301
- Status last verified
- Oct 2026
- First submitted
- 2 Mar 2026
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Multicenter, Vehicle-Controlled, Double-Masked Phase 3 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)
- ClinicalTrials.gov ID
- NCT07453888
- Acronym / sponsor ID
- NGF-NAION-301
- Keywords
- Acute optic neuropathy, Non-Arteritic Anterior Ischemic Optic Neuropathy, NAION, cenegermin, Optic neuropathy, Intranasal
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 272 participants (estimated)
- Lead sponsor
- Dompé Farmaceutici S.p.AIndustry (pharma, biotech, devices)
- Study start
- 1 Jul 2026 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 6 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 3, randomized, multicenter, vehicle-controlled, double-masked study to evaluate the efficacy and safety of intranasal cenegermin compared with vehicle control in adult participants with NAION. Approximately 272 participants who meet all eligibility criteria will be randomly assigned in a 1:1 ratio to receive either cenegermin treatment (Group 1) or the vehicle control (Group 2).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cenegermin | Experimental | Drug: CenegerminParticipants will be administered with cenegermin spray in each nostril. |
| Vehicle | Placebo comparator | Other: VehicleParticipants will be administered with vehicle spray in each nostril. |
Who may qualify
- Ages
- 50 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- A clinical diagnosis of unilateral NAION in the study eye with symptom onset within 14 days prior to the planned date for first dose administration.
- A BCVA score in the study eye of ≥ 15 letters and ≤ 65 letters measured using the ETDRS chart.
- Sufficiently clear ocular media and adequate pupil dilation to enable assessment of the optic nerve and retina in both eyes.
Key exclusion criteria (7 listed)
- Bilateral NAION or sequential NAION with fellow eye involvement within 6 weeks of study eye involvement.
- Clinical evidence of temporal arteritis (giant cell arteritis) signs or symptoms.
- Abnormal laboratory findings suggestive of temporal arteritis (giant cell arteritis), in the absence of a known acute cause
- Pain with eye movement
- Intraocular pressure (IOP) greater than 25 mmHg in the study eye or history of glaucoma in the study eye.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CenegerminCenegermin is administered intranasally. | Drug | Cenegermin |
| VehicleVehicle spray is administered intranasally. | Other | Vehicle |
Primary outcome
Achievement of ≥ 15 letter increase in Best Corrected Visual Acuity (BCVA), assessed in each individual participant, measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart
Time frame: Baseline to Week 24
Improvement in visual acuity defined as a >=15 letter increase in BCVA using ETDRS chart.
5 secondary outcomes
Change from baseline in visual field mean sensitivity in decibels
Time frame: Baseline through Week 24
Change from baseline in BCVA as measured by the ETDRS chart
Time frame: Baseline through Week 24
Change from baseline in visual field mean sensitivity, in a prespecified region consisting of at least 5 separate loci on visual field testing
Time frame: Baseline through Week 24
Change from baseline in ganglion cell layer-inner plexiform layer (GCL-IPL) thickness (micrometer [μm]) on optical coherence tomography (OCT).
Time frame: Baseline through Week 24
Incidence of ocular and non-ocular adverse events
Time frame: Through Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Dompé farmaceutici S.p.A. Via Santa Lucia, 6, 20122 Milan (MI) contact
38 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cenegermin in people with Non-Arteritic Anterior Ischemic Optic Neuropathy, run by Dompé Farmaceutici S.p.A at 38 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, United Kingdom, South Korea, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dompé farmaceutici S.p.A. Via Santa Lucia, 6, 20122 Milan (MI) (+39 02 583 831), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Achievement of ≥ 15 letter increase in Best Corrected Visual Acuity (BCVA), assessed in each individual participant, measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.