What is the mechanism of action of this treatment?

The most common treatments for Thrombocytopenic Purpura (TP) work by either reducing the levels of pathogenic antibodies or increasing platelet production. Efgartigimod, for example, blocks the neonatal Fc receptor (FcRn) to reduce IgG antibody levels, which is important because these antibodies can destroy platelets. Corticosteroids suppress the immune system to lower antibody production, while thrombopoietin receptor agonists like eltrombopag stimulate the production of new platelets. These mechanisms are crucial for managing TP as they help to balance platelet destruction and production, thereby alleviating symptoms and improving patient outcomes.

What side effects are associated with this type of intervention?

Common treatments for Thrombocytopenic Purpura that reduce pathogenic IgG antibodies, such as Efgartigimod, can have side effects including headaches, upper respiratory infections, and infusion-related reactions. There is also a risk of serious infections due to immunosuppression, as well as potential nausea, fatigue, and hypersensitivity reactions.

What prior FDA approvals exist?

The FDA has approved several treatments for Thrombocytopenic Purpura, including thrombopoietin receptor agonists like romiplostim and eltrombopag, which stimulate platelet production. Romiplostim was approved in 2021 for acute radiation syndrome based on animal studies. Eltrombopag has been studied extensively and is used to manage chronic immune thrombocytopenia. These treatments differ from Efgartigimod, which reduces pathogenic IgG antibodies by blocking the neonatal Fc receptor (FcRn), a novel mechanism currently being evaluated in clinical trials.

What other types of research exist?

Promising novel alternatives to ARGX-113 for Thrombocytopenic Purpura patients include thrombopoietin receptor agonists like eltrombopag and romiplostim, which stimulate platelet production. Additionally, investigational therapies such as rituximab, which targets B-cells to reduce antibody production, and fostamatinib, a spleen tyrosine kinase (SYK) inhibitor that reduces immune-mediated platelet destruction, are also potential options.

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Monoclonal Antibodies

Efgartigimod for ITP (ADVANCE+ Trial)

Phase 3

Waitlist Available

Research Sponsored by argenx

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patient has completed a 52-week treatment period

Be older than 18 years old

Must not have

Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects of Efgartigimod may include reactions at the infusion site, headaches, nausea, or allergic responses due to hypersensitivity to its ingredients.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have completed a year-long treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: efgartigimodExperimental Treatment1 Intervention

patients receiving efgartigimod

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

efgartigimod

2019

Completed Phase 3

~140

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Thrombocytopenic Purpura (TP) work by either reducing the levels of pathogenic antibodies or increasing platelet production. Efgartigimod, for example, blocks the neonatal Fc receptor (FcRn) to reduce IgG antibody levels, which is important because these antibodies can destroy platelets. Corticosteroids suppress the immune system to lower antibody production, while thrombopoietin receptor agonists like eltrombopag stimulate the production of new platelets. These mechanisms are crucial for managing TP as they help to balance platelet destruction and production, thereby alleviating symptoms and improving patient outcomes.

Generation of two high affinity anti-mouse FcRn antibodies: Inhibition of IgG recycling in wild type mice and effect in a mouse model of immune thrombocytopenia.