Health TrialsbyHealthPanel

Efgartigimod for the Treatment of Acute Optic Neuritis

ConditionOptic Neuritis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorAnastasia Vishnevetsky, MD, MPH

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Efgartigimod Alfa

    Drug

    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.

  • Placebo

    Drug

    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.

Treatment groups

20 Participants
are divided into 2 treatment groups
Group A: Efgartigmod alfaActive comparator 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Recruitment Rate

Number of enrolled participants per month

Time frame
2 years
2

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
3

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
4

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

Secondary outcomes

1

Retention rate

Percentage of enrolled subjects who remain in the study and do not voluntarily withdraw

Time frame
2 years
2

Screen failure rate

Percentage of participants who fail screening

Time frame
2 years
3

Pre-screen failure rate

Percentage of participants who fail pre-screening

Time frame
2 years
4

Drug adherence rate

Percentage of randomized participants who receive 2 full doses of their assigned study intervention

Time frame
2 years

Other outcomes

1

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
2

Contrast enhancement

Number and proportion of patients enrolled with contrast enhancement of the optic nerves on MRI

Time frame
Day 0
3

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
4

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

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Anastasia Vishnevetsky, MD, MPH

Lead sponsor

Massachusetts General Hospital

Sponsor institution

argenx

Collaborator

Source ClinicalTrials.gov
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