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A Pilot Randomized Trial of Efgartigimod Alfa for the Treatment of Incident Moderate to Severe Acute Optic Neuritis
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
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.
Age
18 - No limit years
Sex
ALL
Healthy Volunteers
No
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Start Date
August 12, 2025
Primary Completion Date
December 1, 2026
Completion Date
July 1, 2027
Last Updated
November 10, 2025
20
ESTIMATED participants
Efgartigimod Alfa
DRUG
Placebo
DRUG
Lead Sponsor
Anastasia Vishnevetsky, MD, MPH
Collaborators
NCT07100990
NCT03401879
Data Source & Attribution
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