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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called efgartigimod alfa to determine if it can help people recover vision more quickly after a first episode of optic neuritis, which inflames the optic nerve and affects sight. The trial aims to discover if efgartigimod alfa is more effective than a placebo (a non-active treatment) and to assess the feasibility of a larger future study. People who have recently experienced vision changes due to optic neuritis might be suitable for this study. Participants will receive standard steroid treatment and have their vision and blood monitored over six months. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that efgartigimod alfa is likely to be safe for humans?

Previous studies have generally found efgartigimod alfa to be well-tolerated. Research shows that common side effects include respiratory infections, headaches, and urinary tract infections, which are usually mild.

A study tracking drug safety over time found that serious cases, involving hospitalizations, occurred in about 53% of cases, with deaths reported in around 9%. However, these figures encompass a wide range of conditions and patient backgrounds, not just optic neuritis.

Long-term safety data from other studies suggest that most patients have tolerated efgartigimod well. This information may reassure those considering joining a trial. Any trial involving efgartigimod will closely monitor participants for safety and side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for optic neuritis?

Efgartigimod alfa is unique because it offers a novel approach to treating optic neuritis by targeting the immune system differently than traditional therapies. Most treatments for optic neuritis, like high-dose corticosteroids, work by broadly suppressing inflammation, but efgartigimod alfa is designed to reduce harmful antibodies by blocking the neonatal Fc receptor (FcRn). This specific mechanism could lead to more targeted treatment with potentially fewer side effects. Researchers are excited because this approach might not only be more effective but also offer faster recovery of vision compared to current options.

What evidence suggests that efgartigimod alfa might be an effective treatment for optic neuritis?

Research has shown that efgartigimod alfa can improve conditions related to the immune system. In some studies, about 49% more patients responded positively to this treatment compared to those who received a placebo. Efgartigimod alfa lowers certain antibodies that mistakenly attack healthy tissue, which is why researchers are testing it for optic neuritis, an inflammation of the optic nerve. Although not yet approved for optic neuritis, results from other conditions suggest potential benefits. In this trial, one group of participants will receive efgartigimod alfa, while another group will receive a placebo. This treatment has shown promise in easing symptoms and reducing the need for steroids in other autoimmune diseases. ^{1 3 5 6 7}

Who Is on the Research Team?

Anastasia Vishnevetsky, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06453694

Efgartigimod for the Treatment of Acute Optic Neuritis The goal of this pilot clinical trial is to test efgartigimod alfa against placebo in adults with first-time optic neuritis (optic nerve inflammation).

2. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK604429/

Clinical Review - Efgartigimod Alfa (Vyvgart) - NCBI Bookshelf It was reported that 49.0% (95% CI, 34.5% to 63.5%) more patients in the efgartigimod alfa group compared with the placebo group achieved a QMG response in ...

3. ctv.veeva.com ctv.veeva.com/study/efgartigimod-for-the-treatment-of-acute-optic-neuritis

Efgartigimod for the Treatment of Acute Optic Neuritis The goal of this pilot clinical trial is to test efgartigimod alfa against placebo in adults with first-time optic neuritis (optic nerve ...

4. frontiersin.org frontiersin.org/journals/neurology/articles/10.3389/fneur.2023.1284444/full

Long-term safety, tolerability, and efficacy of efgartigimod ... Conclusion: Results of ADAPT+ corroborate the substantial clinical improvements seen with efgartigimod in ADAPT and support its long-term safety ...

5. argenx.com argenx.com/news/2024/argenx-highlights-data-showing-patient-impact-across-multiple-im

argenx Highlights Data Showing Patient Impact Across ... Real-world gMG data show that at one-year post VYVGART initiation, 55% of patients reduced corticosteroid use by ≥5 mg/day and 42% of patients ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12041031/

A real-world pharmacovigilance study of efgartigimod alfa ... The most frequently reported serious outcome was hospitalization (53.22%), and death occurred in 270 cases (8.88%).

7. bmjopen.bmj.com bmjopen.bmj.com/content/15/7/e095652

a real-world pharmacovigilance study based on the FAERS ... 8 Early clinical trials have reported that efgartigimod alfa may cause various adverse events (AEs), primarily respiratory infections, headaches and urinary ...