A Phase 2 interventional study of Efgartigimod Alfa and Placebo in Optic Neuritis, sponsored by Anastasia Vishnevetsky, MD, MPH. Recruiting at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-28.
Sponsored by Anastasia Vishnevetsky, MD, MPH · Phase 2, Interventional, and Treatment
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:
Participants will:
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.
88 studies on the registry are indexed under Optic Neuritis; 16 are open to participants now.
This study's planned enrollment of 20 is below the median of 50 across 58 interventional studies indexed under Optic Neuritis.
Browse Optic Neuritis studies →This is the only study on the registry with Anastasia Vishnevetsky, MD, MPH as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
History of 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:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Reproductive potential
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
10 patients will receive efgartigimod alfa All participants will receive standard of care high dose corticosteroids for 3 days with a prednisone taper over 3 weeks.
Drug: Efgartigimod Alfa
10 patients will receive placebo. All participants will receive standard of care high dose corticosteroids for 3 days with a prednisone taper over 3 weeks.
Drug: Placebo
2,016 mg will be administered subcutaneously by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation.
Also known as: Vyvgart Hytrulo
Subcutaneous injection of placebo will be administered by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation.
Recruitment Rate
Number of enrolled participants per month
Time frame: 2 years
Study Adherence Rate
Proportion of randomized participants who receive both doses of assigned study intervention, attend all assigned study visits, and complete at least the high contrast visual acuity, low contrast visual acuity, and Pelli-Robson assessments at all visits
Time frame: 2 years
Change in high contrast visual acuity for effect size and standard deviation estimation
Difference in change in high-contrast visual acuity from baseline to 1 month between groups
Time frame: 1 month
Change in low contrast visual acuity for effect size and standard deviation estimation
Difference in change in low contrast visual acuity (LCVA) (# of letters seen at 2.5% illumination) from baseline to 1 month between groups
Time frame: 1 month
Retention rate
Percentage of enrolled subjects who remain in the study and do not voluntarily withdraw
Time frame: 2 years
Screen failure rate
Percentage of participants who fail screening
Time frame: 2 years
Pre-screen failure rate
Percentage of participants who fail pre-screening
Time frame: 2 years
Drug adherence rate
Percentage of randomized participants who receive 2 full doses of their assigned study intervention
Time frame: 2 years
Full improvement in visual acuity (high contrast)
Proportion of participants with full improvement in high contrast visual acuity
Time frame: 30 days
Full improvement in visual acuity (low contrast)
Proportion of participants with full improvement in low contrast visual acuity
Time frame: 30 days
Personal maximal improvement (high contrast)
Proportion of participants with personal maximal improvement in high contrast visual acuity (defined as individual final visual acuity at 6 months)
Time frame: 30 days
Personal maximal improvement (low contrast)
Proportion of participants with personal maximal improvement in high contrast visual acuity (defined as individual final visual acuity at 6 months)
Time frame: 30 days
Improvement in high contrast visual acuity at 3 months
Difference in change in high contrast visual acuity from baseline to 3 months between groups
Time frame: 3 months
Improvement in high contrast visual acuity at 6 months
Difference in change in high contrast visual acuity from baseline to 6 months between groups
Time frame: 6 months
Rescue treatment
Number and proportion of patients in each arm requiring rescue treatment
Time frame: Day 7
Difference in change in low contrast visual acuity from baseline to 6 months between groups
Difference in change in low contrast visual acuity (# of letters seen at 2.5% illumination) from baseline to 3 months between groups
Time frame: 3 months
Difference in change in low contrast visual acuity from baseline to 6 months between groups
Difference in change in low contrast visual acuity (# of letters seen at 2.5% illumination) from baseline to 6 months between groups
Time frame: 6 months
The number and proportion of participants with low contrast visual acuity of 0
The number and proportion of participants with low contrast visual acuity of 0
Time frame: 30 days
Contrast sensitivity
Difference in change in Pelli-Robson contrast sensitivity score (# of letters seen at 2.5% illumination) from baseline to each assessment time point between groups
Time frame: 6 months
Color Vision
Difference in change in Hardy-Rand-Rittler color vision score from baseline to each assessment time point between groups. Scores range from 0 (lowest) to 6 (highest).
Time frame: 6 months
Visual fields
Difference in change in Humphrey Visual fields score from baseline to each assessment time point between groups. Data derived from automated perimetry are continuous and expressed in decibels. The mean deviation calculated from a Humphrey Visual Field analyzer (24-2 fast paradigm) represents the difference between an individual's test performance and the performance of a normally-sighted control of the same age.
Time frame: 6 months
Vision Related Quality of Life
National Eye Institute Visual Functioning Questionnaire (VFQ-25) scores at baseline, 1 month, 3 months, and 6 months in each arm. Scores range from 0 = worst to 100 = best
Time frame: 6 months
Efgartigimod safety measures
Frequency and type of overall adverse events, treatment-related adverse events, and serious adverse events
Time frame: 6 months
Number and proportion of patients enrolled within 3, 5, 7, and 10 days of visual symptom onset
Number and proportion of patients enrolled within 3, 5, 7, and 10 days of visual symptom onset
Time frame: Day 0
Contrast enhancement
Number and proportion of patients enrolled with contrast enhancement of the optic nerves on MRI
Time frame: Day 0
Median duration (in days) from onset of blurry vision or visual acuity change to randomization
Median duration (in days) from onset of blurry vision or visual acuity change to randomization
Time frame: Day 0
Median duration (in days) from onset of eye pain or headache to randomization
Median duration (in days) from onset of eye pain or headache to randomization
Time frame: Day 0
Number and proportion of overall participants with each diagnosis
Number and proportion of overall participants with a final diagnosis of AQP4+ NMOSD, seronegative NMOSD, MOGAD, MS-related optic neuritis, idiopathic acute optic neuritis, or other diagnosis
Time frame: Day 30
Proportion amongst screened and recruited participants with HCVA of each visual acuity severity
Proportion amongst screened and recruited participants with HCVA worse than logMAR 0.48 (20/60), logMAR 0.7 (20/100), and logMAR 1 (20/200)
Time frame: Day 0
Retinal nerve fiber layer (RNFL) thickness at 1 month between groups
Retinal nerve fiber layer (RNFL) thickness at 1 month between groups
Time frame: 1 month
Retinal nerve fiber layer (RNFL) thickness at 3 months between groups
Retinal nerve fiber layer (RNFL) thickness at 3 months between groups
Time frame: 3 months
Retinal nerve fiber layer (RNFL) thickness at 6 months between groups
Retinal nerve fiber layer (RNFL) thickness at 6 months between groups
Time frame: 6 months
Ganglion cell layer (GCL) thickness at 1 month between groups
Ganglion cell layer (GCL) thickness at 1 month between groups
Time frame: 1 month
Ganglion cell layer (GCL) thickness at 3 months between groups
Ganglion cell layer (GCL) thickness at 3 months between groups
Time frame: 3 months
Ganglion cell layer (GCL) thickness at 6 months between groups
Ganglion cell layer (GCL) thickness at 6 months between groups
Time frame: 6 months
Plan to share: No
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From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Efgartigimod alfa