Actively Recruiting
A Pilot Randomized Trial of Efgartigimod Alfa for the Treatment of Incident Moderate to Severe Acute Optic Neuritis
Led by Anastasia Vishnevetsky, MD, MPH · Updated on 2025-11-10
20
Participants Needed
2
Research Sites
30 weeks
Total Duration
On this page
Sponsors
A
Anastasia Vishnevetsky, MD, MPH
Lead Sponsor
A
argenx
Collaborating Sponsor
AI-Summary
What this Trial Is About
This research aims to evaluate efgartigimod alfa compared to placebo in adults experiencing their first episode of optic neuritis, which is inflammation of the optic nerve. The study investigates whether efgartigimod alfa is feasible to use for optic neuritis, if conducting a larger trial is possible, and how the treatment affects the speed and extent of vision recovery. This is a pilot, randomized, placebo-controlled clinical trial led by Anastasia Vishnevetsky, MD, MPH. Participants are randomly assigned to one of two groups: one group receives efgartigimod alfa, and the other receives a placebo. Both groups also receive standard care treatment consisting of high-dose corticosteroids for 3 days followed by a prednisone taper over 3 weeks. Efgartigimod alfa or placebo is given by subcutaneous injection on Day 0 and Day 3. If needed, rescue therapy with plasma exchange is available based on evaluation results on Day 7. Throughout the 6-month study period, participants will have multiple visits where their vision is tested and blood samples are taken. They will also answer questions about their vision. Researchers will measure changes in high and low contrast visual acuity at 1 month and up to 6 months, monitor safety, and track adherence and retention. The study includes standard care and careful monitoring to understand the treatment's effects on vision recovery and quality of life.
CONDITIONS
Brief Title
Efgartigimod for the Treatment of Acute Optic Neuritis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed and dated informed consent form
- Willingness to comply with all study procedures and availability for the study duration
- Adults aged 18 years or older
- Diagnosed with first episode of optic neuritis confirmed by MRI
- Onset of optic neuritis vision changes within 10 days of enrollment
- Worse affected eye has high contrast visual acuity of logMAR 0.48 (20/60) or worse
- For females of reproductive potential: negative pregnancy test or use of effective contraception
- For males of reproductive potential: use of condoms or effective contraception methods
You will not qualify if you...
- Current pregnancy or breastfeeding
- Allergic reactions or intolerance to efgartigimod, methylprednisolone, prednisone, or gadolinium
- Previous optic neuropathy diagnosis before current episode
- Systemic disease other than MS, NMOSD, or MOGAD linked to optic neuritis
- Receiving or planned systemic immunomodulatory or immunosuppressive therapy within 3 weeks of treatment
- Known diagnosis of CNS demyelinating disease prior to current attack
- Significant eye diseases affecting vision before optic neuritis
- Alternative causes for vision changes detected on eye exams
- Participation in another investigational treatment study within 2 months
- Contraindications to MRI or plasma exchange
- More than 3 days of high-dose steroids given for current optic neuritis episode before randomization
- Known HIV or common variable immunodeficiency
- History of malignancy unless cured and without recurrence for at least 1 year, except certain treated skin and cervical cancers
- Active or chronic infections that are uncontrolled
- Recent major surgery within 1 month or planned surgery during study
- Any condition judged by investigator as risky or unsuitable for study participation
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 3 weeks
Participants receive either efgartigimod alfa or placebo by subcutaneous injection on Day 0 and Day 3 along with standard of care high dose corticosteroids for 3 days followed by a prednisone taper over 3 weeks. Rescue therapy with plasma exchange may be provided based on Day 7 evaluation.
Visits on Day 0, Day 3, and Day 7 with additional visits during prednisone taper
Duration - Up to 6 months
Participants are monitored for changes in visual acuity and safety outcomes after treatment ends.
Periodic visits up to 6 months post-treatment
Trial Site Locations
Total: 2 locations
1
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States, 02114
Actively Recruiting
2
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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