Efgartigimod for Optic Neuritis

(PET-AON Trial)

Recruiting at 1 trial location

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Anastasia Vishnevetsky, MD, MPH

Must be taking: Steroids

Must not be taking: Immunosuppressive therapy

Disqualifiers: Pregnancy, Allergies, CNS disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

Duration: 4 weeks

20 Participants Needed

Trial Summary

What is the purpose of 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

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot be on systemic immunomodulatory or immunosuppressive therapy at the time of enrollment or within 1 month of treatment.

How does the drug Efgartigimod Alfa differ from other treatments for optic neuritis?

Efgartigimod Alfa is unique because it is designed to reduce the levels of harmful antibodies in the body, which may help in conditions like optic neuritis associated with MOG-IgG. This mechanism is different from traditional treatments like steroids, which primarily reduce inflammation. ^{1 2 3 4 5}

Research Team

Anastasia Vishnevetsky, MD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

This trial is for adults experiencing their first episode of optic neuritis, which is inflammation of the optic nerve. Participants must be willing to undergo vision and blood tests, answer questions about their vision, receive standard steroid treatment, and attend periodic visits over a six-month period.

Inclusion Criteria

Provision of signed and dated informed consent form

Stated willingness to comply with all study procedures and availability for the duration of the study

I am using effective birth control methods if I am capable of fathering a child.

Exclusion Criteria

I had a major surgery recently.

Current pregnancy or lactation

I cannot undergo MRI or plasma exchange due to health reasons.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod alfa or placebo in addition to standard of care high dose corticosteroids for 3 days with a prednisone taper over 3 weeks

4 weeks

Weekly visits for 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of visual acuity and contrast sensitivity

6 months

Periodic visits over 6 months

Rescue Treatment (if needed)

Option for rescue therapy with plasma exchange for participants with poor therapeutic response

Treatment Details

Interventions

• Efgartigimod Alfa

Trial Overview The study is testing efgartigimod alfa against a placebo in treating acute optic neuritis. It aims to determine if efgartigimod alfa can be feasibly used for this condition and whether it's more effective than a placebo in restoring vision quickly and significantly.

Participant Groups

Group I: Efgartigmod alfa Active Control 1 Intervention

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.

Group II: Placebo Placebo Group 1 Intervention

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.

Who Is Running the Clinical Trial?

Anastasia Vishnevetsky, MD, MPH

Lead Sponsor

Trials

1

Recruited

20+

argenx

Industry Sponsor

Trials

76

Recruited

11,500+

Tim Van Hauwermeiren

argenx

Chief Executive Officer since 2008

B.Sc. and M.Sc. in Bioengineering from Ghent University, Executive MBA from The Vlerick School of Management

Dr. Peter Ulrichts

argenx

Chief Medical Officer since 2023

MD from Maastricht University, PhD in Molecular Immunology from Maastricht University

References

1. United States pubmed.ncbi.nlm.nih.gov

Clinical Characteristics and Treatment of MOG-IgG-Associated Optic Neuritis. [2020]

2. England pubmed.ncbi.nlm.nih.gov

Time to steroids impacts visual outcome of optic neuritis in MOGAD. [2023]

3. Germany pubmed.ncbi.nlm.nih.gov

Neuro-ophthalmological Presentation of Optic Neuritis in Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Neuromyelitis optica spectrum disorder and myelin oligodendrocyte glycoprotein associated disorder-optic neuritis: a comprehensive review of diagnosis and treatment. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Relapsing MRI-negative myelitis associated with myelin-oligodendrocyte glycoprotein autoantibodies: a case report. [2022]