Can a drug for muscle weakness help save sight in optic neuritis?
NCT ID NCT06453694
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This pilot study tests efgartigimod alfa (Vyvgart Hytrulo) against placebo in 20 adults having their first episode of moderate to severe optic neuritis, an inflammation of the optic nerve that can cause vision loss. Participants get two injections over a week, plus standard steroid treatment, and are followed for 6 months. The main goal is to see if a larger trial is feasible and whether the drug improves vision recovery.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- efgartigimod alfa (Vyvgart Hytrulo)
- What this could lead to
- If this works, it could point toward a new treatment to help people recover vision faster after optic neuritis.
- What could go wrong
- This is a very small, early pilot trial with only 20 people. It's mainly checking if a larger study is possible, not yet proving the drug works. Results may not apply to everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 20 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2025
- Expected to finish
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Jul 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Provision of signed and dated informed consent form 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Adults aged 18 years or older 4. 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 5. 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. 6. 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. 7. 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 8. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner Exclusion Criteria: 1. Current pregnancy or lactation 2. Known allergic reactions or intolerance to efgartigimod, methylprednisolone, prednisone, or gadolinium or any of their components 3. Known diagnosis of optic neuropathy preceding the current episode of optic neuritis 4. Evidence of a systemic disease other than MS, NMOSD, or MOGAD that might be associated with the optic neuritis 5. 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. 6. Known diagnosis of CNS demyelinating disease (MS, NMOSD, MOGAD) prior to present attack. 7. 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. 8. Alternative explanation for visual changes detected on fundoscopic exam and slit lamp examination. 9. Enrollment in another clinical study involving an investigational treatment given within 2 months of enrollment in the present study. 10. Contraindication to MRI or plasma exchange 11. Has received \>3 days of high-dose steroids (IV or PO) for the treatment of the current episode of acute optic neuritis by the time of randomization. Randomization may occur at the latest on the next day after completion of 3rd dose of steroids. 12. Known HIV disease or common variable immunodeficiency 13. History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥1 year before the first administration of IMP. Adequately treated participants with the following cancers may be included at any time: 1. Basal cell or squamous cell skin cancer 2. Carcinoma in situ of the cervix 3. Carcinoma in situ of the breast 4. Incidental histological finding of prostate cancer (TNM stage T1a or T1b) 14. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection 15. Clinically significant recent major surgery (within 1 month of screening), or intends to have surgery during the study 16. Any conditions or circumstances that in the opinion of the investigator may put the participant at undue risk, confound the results of the study, or otherwise make the participant unsuitable for the study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
2 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Massachusetts Eye and Ear Infirmary
RECRUITINGBoston, Massachusetts, 02114, United States
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Massachusetts General Hospital
RECRUITINGBoston, Massachusetts, 02114, United States
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Other studies related to the condition(s) this trial covers.
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