Can a drug for muscle weakness help save sight in optic neuritis?

NCT ID NCT06453694

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 20 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2025

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

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

18 years and older

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    2 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. 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

  • Massachusetts Eye and Ear Infirmary

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

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