NCT06453694

Efgartigimod for the Treatment of Acute Optic Neuritis

A Pilot Randomized Trial of Efgartigimod Alfa for the Treatment of Incident Moderate to Severe Acute Optic Neuritis

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Anastasia Vishnevetsky, MD, MPH · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this pilot clinical trial is to test efgartigimod alfa against placebo in adults with first-time optic neuritis (optic nerve inflammation). The main questions it aims to answer are: * Is it feasible to use efgartigimod alfa for optic neuritis? * Is it feasible to run a larger trial testing efgartigimod alfa in optic neuritis? * Does efgartigimod alfa work better than placebo in improving how quickly and how much vision returns? Participants will: * have their vision and blood tested * be asked questions about their vision * will receive standard of care treatment with steroids regardless of whether they are receiving efgartigimod alfa or not * will have periodic visits over 6 months

Detailed description

This study is designed as a pilot, single-site, randomized, placebo-controlled, 2-arm, parallel-group clinical trial comparing efgartigimod alfa in addition to standard of care (IV steroids with a standardized oral taper) to standard of care with placebo, with an option for rescue therapy with plasma exchange for all participants in the case of poor therapeutic response.

Conditions

• Optic Neuritis

Interventions

Type	Name	Description
DRUG	Efgartigimod Alfa	2,016 mg will be administered subcutaneously by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation.
DRUG	Placebo	Subcutaneous injection of placebo will be administered by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation.

Timeline

• Start date: 2025-08-12
• Primary completion: 2026-12-01
• Completion: 2027-07-01
• First posted: 2024-06-12
• Last updated: 2025-11-10

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study