Efficacy and Safety of Intranasal Cenegermin in Adult Participants With Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)

Dompé

Status and phase: Not yet enrolling; Phase 3

Conditions: Non-Arteritic Anterior Ischemic Optic Neuropathy

Treatments: Other: Vehicle; Drug: Cenegermin

Study type: Interventional

Funder types: Industry

Identifiers

NCT07453888

NGF-NAION-301

Details and patient eligibility

About

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).

Enrollment: 272 estimated patients

Sex: All

Ages: 50 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

1. 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.
2. A BCVA score in the study eye of ≥ 15 letters and ≤ 65 letters measured using the ETDRS chart.
3. Sufficiently clear ocular media and adequate pupil dilation to enable assessment of the optic nerve and retina in both eyes.

Key Exclusion Criteria:

1. Bilateral NAION or sequential NAION with fellow eye involvement within 6 weeks of study eye involvement.
2. Clinical evidence of temporal arteritis (giant cell arteritis) signs or symptoms.
3. Abnormal laboratory findings suggestive of temporal arteritis (giant cell arteritis), in the absence of a known acute cause
4. Pain with eye movement
5. Intraocular pressure (IOP) greater than 25 mmHg in the study eye or history of glaucoma in the study eye.
6. 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 design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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272 participants in 2 patient groups, including a placebo group

Cenegermin

Experimental group

Description:

Participants will be administered with cenegermin spray in each nostril.

Treatment:

Drug: Cenegermin

Vehicle

Placebo Comparator group

Description:

Participants will be administered with vehicle spray in each nostril.

Treatment:

Other: Vehicle