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
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
20
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.
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.
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
RECRUITINGMassachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGRecruitment 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
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