Efgartigimod for the Treatment of Acute Optic Neuritis (NCT06453694) | Clinical Trial Compass
RecruitingPhase 2
Efgartigimod for the Treatment of Acute Optic Neuritis
United States20 participantsStarted 2025-08-12
Plain-language 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
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Provision of signed and dated informed consent form
✓. Stated willingness to comply with all study procedures and availability for the duration of the study
✓. Adults aged 18 years or older
✓. Diagnosed with a first episode of optic neuritis, based on clinical presentation (i.e. typical features such as pain with eye movements, color vision changes, subacute presentation, and visual acuity loss) and confirmed by contrast enhancement or T2 hyperintensity of the optic nerve on MRI brain or orbits using a 1.5T MRI scanner or greater
✓. Onset of optic neuritis-related vision changes (does not include headache, eye pain, or pain with eye movements), as defined by decreased visual acuity, subjectively reported blurred vision, or optic nerve enhancement on MRI brain or orbits, within 10 days (inclusive) of enrollment. If optic neuritis is bilateral, then enrollment must occur within 10 days of vision changes in the first affected eye.
✓. Best-corrected high contrast visual acuity (HCVA) in the worse affected eye on the Early Treatment Diabetic Retinopathy Study (ETDRS) eye chart of logMAR 0.48 (20/60) or worse.
✓. For females of reproductive potential: negative urine or serum pregnancy test at screening or use of highly effective contraception for at least 1 month prior to screening and agreement to use such a method during study participation and for an additional 8 weeks after the end of efgartigimod administration
What they're measuring
1
Recruitment Rate
Timeframe: 2 years
2
Study Adherence Rate
Timeframe: 2 years
3
Change in high contrast visual acuity for effect size and standard deviation estimation
Timeframe: 1 month
4
Change in low contrast visual acuity for effect size and standard deviation estimation
✓. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
Exclusion criteria
✕. Current pregnancy or lactation
✕. Known allergic reactions or intolerance to efgartigimod, methylprednisolone, prednisone, or gadolinium or any of their components
✕. Known diagnosis of optic neuropathy preceding the current episode of optic neuritis
✕. Evidence of a systemic disease other than MS, NMOSD, or MOGAD that might be associated with the optic neuritis
✕. Receiving systemic immunomodulatory or immunosuppressive therapy at the time of enrollment or planned receipt within 3 weeks of treatment. Initiation of immunotherapy more than 3 weeks after the second dose of efgartigimod is not an exclusion criterion and is permitted.
✕. Known diagnosis of CNS demyelinating disease (MS, NMOSD, MOGAD) prior to present attack.
✕. Any visually-significant ocular pathology (i.e. retinal problems, cataracts, glaucoma etc.) in the affected eye that led to known best-corrected visual acuity deficits in participants prior to onset of optic neuritis. Congenital color-blindness is not disqualifying.
✕. Alternative explanation for visual changes detected on fundoscopic exam and slit lamp examination.