Efgartigimod PH20 SC for Bioequivalence Study

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how efgartigimod PH20 SC, a treatment for autoimmune conditions, behaves in the body when administered through two different types of syringes. The goal is to determine any differences between using a prefilled syringe and a vial and syringe for medication delivery. The study targets healthy adults who meet specific health criteria, such as a body mass index (BMI) between 18 and 30 and no autoimmune diseases. Participants should also have no history of major health issues, such as recent cancer or significant infections. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research shows that efgartigimod PH20 SC is generally safe for people. Studies on the prefilled syringe version have found it safe for patients with conditions like primary Sjögren's Disease, with no serious side effects reported. Data on the vial and syringe version also indicate safety, as studies have used the treatment without major safety concerns. Current research suggests that both versions of efgartigimod PH20 SC are safe for people. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about Efgartigimod PH20 SC because it offers a new, convenient way to deliver treatment through a prefilled syringe, making it easier and potentially more comfortable for patients compared to the traditional vial and syringe method. Unlike existing treatments that may require more complicated administration, this method simplifies the process, which can improve patient adherence and overall experience. The new delivery approach doesn't change the active ingredient, but it could make a significant difference in the practicality and accessibility of treatment.

What evidence suggests that this trial's treatments could be effective?

Research has shown that efgartigimod PH20 SC effectively treats certain autoimmune diseases. One study demonstrated that it reduced symptoms in people with generalized myasthenia gravis, a condition that weakens muscles, as effectively as an intravenous version. Another study found it helped lower the chance of relapse in people with chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder. These studies also confirmed that the treatment is safe and well-tolerated over time. This trial will compare two methods of administering efgartigimod PH20 SC: one using a vial and syringe, and the other using a prefilled syringe. This suggests that efgartigimod PH20 SC could be a promising treatment option for these conditions. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod PH20 SC

How Is the Trial Designed?

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

Published Research Related to This Trial

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, 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 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 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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04598477

NCT04598477 | A Study to Assess the Long-term Safety ... This was a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39227284/

A phase 3 randomized noninferiority study (ADAPT-SC) ... These findings support noninferiority between efgartigimod PH20 SC 1000 mg and efgartigimod IV 10 mg/kg, as well as long-term safety, tolerability, and ...

3. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/aan-2025/Long-Term-Safety-and-Efficacy-of-Subcutaneous-Efgartigimod-PH20-in-Adult-Participants-With-Generalized-Myasthenia-Gravis-Interim-Results-of-the-ADAPT-S.pdf

Interim Results of the ADAPT-SC+ Study eMost frequent AEs occurring in >10% of participants receiving efgartigimod PH20 SC in either data cut. ▫ Participants experiencing ...

4. thelancet.com thelancet.com/journals/laneur/article/PIIS1474-4422(24)00309-0/fulltext

Safety, tolerability, and efficacy of subcutaneous ... ADHERE showed the efficacy of subcutaneous efgartigimod PH20 in reducing the risk of relapse versus placebo in people with CIDP who responded to treatment.

5. neurology.org neurology.org/doi/10.1212/WNL.0000000000210678

Interim Results of the ADAPT-SC+ Study (P1-11.005) Results: As of December 2022, 179 participants received ≥1 dose of efgartigimod PH20 SC, with a mean (SD) study duration of 413 (105) ...

6. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/eular-june-2025/Efficacy%20and%20Safety%20of%20Efgartigimod%20PH20%20Subcutaneous%20by%20Prefilled%20Syringe%20in%20Adults%20With%20Primary%20Sjogrens%20Disease%20A%20Phase%203_%20Randomized_%20Double-Blind_%20Placebo-Controlled_%20Multicenter%20Trial%20With%20.pdf

Efficacy and Safety of Efgartigimod PH20 Subcutaneous by ... ... Safety, and Tolerability of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Patients With pSjD (NCT06684847). ≤4 weeks ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT06307613

NCT06307613 | A Study of Efgartigimod PH20 SC Given ... This study aims to evaluate the efficacy, safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of efgartigimod PH20 SC

8. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/mgfa-2024/Design%20of%20a%20Phase%203%20Randomized,%20Double-Blinded,%20Placebo-Controlled%20Study%20Evaluating%20the%20Efficacy%20and%20Safety%20of%20Subcutaneous%20Efgartigimod%20PH20%20Adm.pdf

Design of a Phase 3 Randomized, Double-Blinded, ... This phase 3 trial will provide important data on the efficacy and safety of efgartigimod PH20 SC prefilled syringe in the treatment of oMG, including.

9. clinicaltrials.gov clinicaltrials.gov/study/NCT06684847

NCT06684847 | A Study of the Efficacy and Safety ... The main purpose of the proposed study is to evaluate the efficacy of efgartigimod PH20 SC in patients with moderate-to-severe Primary Sjögren's Disease ...

10. argenx.com argenx.com/news/2024/argenx-presents-new-efgartigimod-data-at-eular-2025-highlighting

argenx Presents New Efgartigimod Data at EULAR 2025 ... Efficacy and safety of efgartigimod PH20 subcutaneous by prefilled syringe in adults with Sjögren's disease: A Phase 3, randomized, double ...