A Phase 1 Bioequivalence Study of Efgartigimod PH20 SC Administered Via a Prefilled Syringe Versus a Vial+Syringe Presentation in Healthy Adults

Recruiting at 1 trial location

Overseen By Sabine s Coppieters, MD

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: argenx

Must not be taking: Concomitant medications

Disqualifiers: Autoimmune disease, Malignancy, Active infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

120 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing how a drug called efgartigimod behaves in the blood when given to healthy people. The drug is given either through a prefilled syringe or a vial and syringe to see if there is any difference in how it is processed by the body.

Will I have to stop taking my current medications?

Yes, you will need to stop taking your current medications, except for oral contraceptives or occasional acetaminophen, to participate in this trial.

What data supports the effectiveness of the drug Efgartigimod PH20 SC?

Efgartigimod has been shown to be effective in treating generalized myasthenia gravis (a condition causing muscle weakness) by significantly reducing disease burden and improving muscle strength and quality of life. It has also been studied for other autoimmune diseases, showing potential benefits by reducing harmful antibodies. ^{1 2 3 4 5}

How is the drug Efgartigimod PH20 SC different from other treatments for autoimmune conditions?

Efgartigimod PH20 SC is unique because it is a first-in-class drug that targets the neonatal Fc receptor, reducing the levels of harmful antibodies in the body. This mechanism is different from other treatments, which often focus on suppressing the immune system more broadly. Additionally, it is available in both intravenous and subcutaneous forms, offering flexibility in administration. ^{1 2 3 4 6}

Eligibility Criteria

Inclusion Criteria

You are between 18 and 55 years old, which is the legally accepted age range for participating in a clinical study.

You are able to sign a consent form and comply with the study protocol.

Women Of Child-Bearing Potential must affirmatively demonstrate a negative serum hCG pregnancy test at screening and a negative urine hCG pregnancy test at baseline prior to receiving IMP in accordance with regional regulations.

Exclusion Criteria

Has a known autoimmune disease or any medical condition that, in the investigator's judgment, would interfere with an accurate assessment of clinical symptoms or puts the participant at undue risk

Has a history of malignancy, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years before the administration of IMP. Adequately-treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological findings of prostate cancer.

Has a clinically significant active infection that is not sufficiently resolved in the investigator's opinion.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of efgartigimod PH20 SC via either a prefilled syringe or a vial + syringe

1 day

Follow-up

Participants are monitored for pharmacokinetics and safety, including PK parameters and incidence of antidrug antibodies

57 days

Treatment Details

Interventions

• Efgartigimod PH20 SC

Participant Groups

Group I: Efgartigimod PH20 SC - prefilled syringe Experimental Treatment 1 Intervention

efgartigimod PH20 SC administered by a prefilled syringe

Group II: Efgartigimod PH20 SC - vial + syringe Active Control 1 Intervention

efgartigimod PH20 SC administered by a vial + syringe

Efgartigimod PH20 SC is already approved in European Union, United States, Japan, China for the following indications:

Approved in European Union as VYVGART for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive

Approved in United States as VYVGART Hytrulo for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
• Chronic inflammatory demyelinating polyneuropathy (CIDP)

Approved in Japan as VYVDURA for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive

Approved in China as Efgartigimod alfa injection (subcutaneous injection) for:

• Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive

Who Is Running the Clinical Trial?

argenx

Lead 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

Findings from Research

Efgartigimod alfa is the first neonatal Fc receptor antagonist approved for treating generalized myasthenia gravis (gMG), showing significant and rapid improvements in muscle strength and quality of life in a phase 3 trial with a placebo group.

The treatment was generally well tolerated, with most side effects being mild to moderate, indicating a favorable safety profile for patients with gMG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. Heo, YA.[2023]

Efgartigimod was found to be safe and well-tolerated in a phase 2 study involving 24 patients with generalized myasthenia gravis, with no serious adverse events reported.

The treatment led to a significant reduction in pathogenic IgG autoantibodies and resulted in a rapid and long-lasting improvement in disease symptoms for 75% of patients, suggesting its potential as an effective therapy for myasthenia gravis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. Howard, JF., Bril, V., Burns, TM., et al.[2020]

Efgartigimod, a first-in-class neonatal Fc receptor antagonist, received its first approval in the USA in December 2021 for treating generalized myasthenia gravis in adults who are positive for anti-acetylcholine receptor antibodies.

The drug is also being evaluated for other autoimmune diseases and has been approved in Japan for generalized myasthenia gravis patients regardless of antibody status, indicating its potential broad application in autoimmune conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. Heo, YA.[2022]

Efgartigimod is a promising new treatment for primary immune thrombocytopenia (ITP) that works by binding to the neonatal Fc receptor (FcRn), which leads to increased degradation of IgG antibodies that cause platelet destruction.

This therapy offers a novel mechanism of action compared to existing treatments, potentially providing an effective option for patients who may not respond well to traditional therapies like corticosteroids or IVIgG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod alfa for the treatment of primary immune thrombocytopenia. Broome, C.[2023]

In a Phase 2 study involving 38 patients with primary immune thrombocytopenia (ITP), efgartigimod, an FcRn antagonist, was well tolerated and showed a favorable safety profile, consistent with earlier Phase 1 data.

Efgartigimod treatment led to a significant reduction in total IgG levels and clinically relevant increases in platelet counts, with 46% of patients achieving a platelet count of ≥50 × 10^9/L compared to 25% on placebo, indicating its potential as a novel treatment for ITP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Newland, AC., Sánchez-González, B., Rejtő, L., et al.[2021]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. [2023]

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

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. [2020]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. [2022]

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

Efgartigimod alfa for the treatment of primary immune thrombocytopenia. [2023]

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

Effect of FcRn antagonism on protective antibodies and to vaccines in IgG-mediated autoimmune diseases pemphigus and generalised myasthenia gravis. [2022]

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

Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. [2021]