Efgartigimod for ITP

(ADVANCE+ Trial)

Not currently recruiting at 114 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called efgartigimod, an immunotherapy, to evaluate its effectiveness and safety for people with primary Immune Thrombocytopenia (ITP). In ITP, the immune system attacks and destroys platelets, causing easy bruising and bleeding. Participants will receive efgartigimod, and researchers will monitor their progress over time. The trial seeks adults who have completed a previous related study with efgartigimod. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for ITP.

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.

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

Research has shown that efgartigimod is generally well-tolerated by patients. Studies have found that it can help increase platelet counts in people with chronic immune thrombocytopenia (ITP), a condition characterized by a lower than normal platelet count. This increase suggests the treatment works effectively without causing serious issues.

Efgartigimod is already approved for use in other conditions, such as generalized myasthenia gravis (a disorder that causes muscle weakness) and ITP in Japan. This approval indicates it has undergone safety testing for these conditions.

Previous studies have reported no major safety concerns. While all treatments can have side effects, current data suggests that efgartigimod is safe for use in humans. ^{1 2 3 4 5}

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

Unlike the standard treatments for Immune Thrombocytopenia (ITP), which often include steroids and immunosuppressants, efgartigimod offers a novel approach by targeting the neonatal Fc receptor (FcRn). This mechanism is unique because it reduces the lifespan of autoantibodies that attack and destroy platelets, potentially offering a more direct and efficient way to manage the condition. Researchers are excited about efgartigimod because it has the potential to improve platelet counts more rapidly and with fewer side effects compared to traditional therapies.

What evidence suggests that efgartigimod might be an effective treatment for ITP?

Research has shown that efgartigimod, which participants in this trial will receive, effectively treats patients with primary immune thrombocytopenia (ITP). Studies have demonstrated that it significantly increased long-lasting platelet counts in patients with chronic ITP compared to those who received a placebo. Specifically, the ADVANCE trial found that more patients taking efgartigimod experienced better platelet responses, crucial for reducing the risk of bleeding. This suggests that efgartigimod can help manage ITP by increasing platelet levels, leading to better health outcomes for patients. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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]

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 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 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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06544499

NCT06544499 | A Study to Assess the Efficacy and Safety ... The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP).

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04188379

NCT04188379 | A Study to Assess the Efficacy and Safety ... This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37778358/

Efficacy and safety of the neonatal Fc receptor inhibitor ... Efgartigimod significantly increased sustained platelet count responses compared with placebo in patients with chronic immune thrombocytopenia.

4. argenx.com argenx.com/news/2022/argenx-announces-positive-phase-3-data-advance-trial-vyvgartr-efgartigimod-alfa-fcab-adults

argenx Announces Positive Phase 3 Data from ADVANCE ... ADVANCE met its primary endpoint demonstrating that a higher proportion of chronic ITP patients receiving VYVGART achieved a sustained platelet count response ...

5. argenx.com argenx.com/news/2025/press-release-3176635

argenx Presents New Data at AANEM and MGFA ... “The ADAPT SERON trial showed that efgartigimod generated tangible improvements in daily functioning, marking an important advancement for the ...

6. ema.europa.eu ema.europa.eu/en/documents/variation-report/vyvgart-h-c-005849-ii-0020-epar-assessment-report-variation_en.pdf

Vyvgart, INN: efgartigimod alfa - EMA ... efgartigimod PH20 SC safety data from participants with CIDP. It also allows for the evaluation of long-term longitudinal safety data and data ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04188379

NCT04188379 | A Study to Assess the Efficacy and Safety ... This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP ...