Efgartigimod for ITP

(ADVANCE+ Trial)

Recruiting at 86 trial locations

Overseen By Antonio Guglietta, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: argenx

Must not be taking: Anti-CD20, Romiplostim, Monoclonal antibodies, others

Disqualifiers: Pregnancy, Lactation, Hypersensitivity, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

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

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

101 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing ARGX-113, a medication for adults with primary ITP, a condition where the immune system attacks platelets. The medication aims to calm the immune system to prevent it from attacking these important blood cells.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but certain medications like anti-CD20 therapy and romiplostim are not allowed. It's best to discuss your current medications with the trial team.

What data supports the effectiveness of the drug Efgartigimod for treating ITP?

Research shows that Efgartigimod, when used in patients with ITP, led to a significant increase in platelet counts and reduced bleeding. In a study, 46% of patients receiving Efgartigimod achieved a platelet count of at least 50 x 10^9/L on multiple occasions, compared to 25% on placebo, indicating its potential effectiveness. ^{1 2 3 4 5}

Is efgartigimod safe for humans?

Efgartigimod has been generally well tolerated in clinical trials for conditions like immune thrombocytopenia and myasthenia gravis, with most side effects being mild to moderate. ^{1 2 4 5 6}

How is the drug efgartigimod different from other treatments for ITP?

Efgartigimod is unique because it targets the neonatal Fc receptor (FcRn) to reduce levels of IgG antibodies, which are responsible for platelet destruction in ITP. This mechanism is different from other treatments like corticosteroids or IVIgG, offering a novel approach to managing the condition. ^{1 2 4 5 6}

Eligibility Criteria

Adults with primary ITP, a condition causing low platelet count, can join this trial if they've completed prior phases. Women must use effective contraception and have a negative pregnancy test. Participants should understand the trial demands and not be on excluded medications or treatments.

Inclusion Criteria

I have completed a year-long treatment.

Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits)

I completed the 24-week ARGX-113-1801 trial.

Exclusion Criteria

I am not taking any restricted medications like anti-CD20 therapy or live vaccines.

Use of any other investigational drug or participation in any other investigational trial

You have a history of being very sensitive or allergic to any of the ingredients in efgartigimod.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod 10 mg/kg intravenously

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Efgartigimod

Trial Overview The study is testing Efgartigimod's long-term effectiveness and safety for treating primary ITP in adults. It's an open-label phase 3 trial where everyone receives the drug to see how well it works over time.

Participant Groups

Group I: efgartigimod Experimental Treatment 1 Intervention

patients receiving efgartigimod

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

Approved in European Union as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)

Approved in United States as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)
• Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Approved in Canada as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)

Approved in Japan as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)

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

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]

In a phase 3 study involving 131 adults with chronic primary immune thrombocytopenia, efgartigimod significantly improved sustained platelet count responses compared to placebo, with 22% of patients achieving the primary endpoint versus 5% in the placebo group.

Efgartigimod was well tolerated, with most adverse events being mild to moderate, indicating a favorable safety profile for patients who had previously undergone multiple treatments for their condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. Broome, CM., McDonald, V., Miyakawa, Y., et al.[2023]

In a study of 64 patients with chronic immune thrombocytopenic purpura (ITP), 81.3% achieved a significant increase in platelet counts after receiving 10% intravenous immunoglobulin (IVIG) treatment, indicating its efficacy.

The treatment was found to be safe, with 92.2% of patients experiencing only mild to moderate adverse events, suggesting a favorable safety profile for both adults and children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of intravenous immunoglobulin (Flebogamma® 10% DIF) in patients with immune thrombocytopenic purpura. Apte, S., Navarro-Puerto, J., Damodar, S., et al.[2019]

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]

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]

References

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

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

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

Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Safety and efficacy of intravenous immunoglobulin (Flebogamma® 10% DIF) in patients with immune thrombocytopenic purpura. [2019]

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

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

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

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

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

Efgartigimod: First Approval. [2022]