What side effects are associated with this type of intervention?

Common treatments for Thrombocytopenic Purpura (ITP) include corticosteroids, intravenous immunoglobulin (IVIG), and thrombopoietin receptor agonists like romiplostim and eltrombopag. Corticosteroids can cause weight gain, hypertension, and increased risk of infections. IVIG may lead to headaches, fever, and allergic reactions. Thrombopoietin receptor agonists, such as romiplostim and eltrombopag, can cause headaches, fatigue, and increased risk of thromboembolic events. Specific to FcRn antagonists like Efgartigimod PH20, potential side effects may include infections and infusion-related reactions, although detailed data is still being evaluated in ongoing trials.

What prior FDA approvals exist?

The FDA has approved several treatments for Thrombocytopenic Purpura, including thrombopoietin receptor agonists like romiplostim and eltrombopag. Romiplostim is effective in raising platelet counts and is used in patients with chronic immune thrombocytopenia. Eltrombopag has shown similar efficacy and is also approved for this condition. These treatments work by stimulating platelet production, unlike Efgartigimod PH20, which is an FcRn antagonist currently under study. FcRn antagonists represent a different therapeutic approach by modulating the immune system to reduce the destruction of platelets.

What other types of research exist?

Promising novel alternatives to Efgartigimod PH20 for Thrombocytopenic Purpura patients include thrombopoietin-receptor agonists like eltrombopag and romiplostim, which have shown efficacy in clinical trials. Additionally, monoclonal antibodies such as eculizumab and its second-generation version, ravulizumab, are being explored for their potential benefits in immune-related conditions. These alternatives offer different mechanisms of action and have demonstrated positive outcomes in recent studies.

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Monoclonal Antibodies

Efgartigimod for Low Platelet Count (ADVANCE SC+ 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

Participant has completed a 52-week treatment period

Be older than 18 years old

Must not have

Introduction or continuation of nonpermitted medications during the ARGX-113-2004 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 a new injection treatment for adults whose immune systems destroy their own platelets. The treatment aims to calm the immune system to stop it from attacking these important blood cells. Romiplostim promotes platelet production and has been used in the treatment of this condition.

Who is the study for?

This trial is for adults with primary immune thrombocytopenia who completed a previous 24-week study and can commit to another 52 weeks. They must understand the trial, consent to procedures, attend visits, use contraception if applicable, and not be pregnant or breastfeeding.

What is being tested?

The safety and effectiveness of efgartigimod PH20 SC (a subcutaneous injection) are being tested in patients with low platelet counts due to primary immune thrombocytopenia over an additional year following a prior study.

What are the potential side effects?

Potential side effects may include allergic reactions to efgartigimod PH20 SC or its ingredients. Specific side effects are not listed but would typically relate to the body's response to new medications.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have completed a 52-week treatment period.

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

Side effects data

From 2023 Phase 3 trial • 222 Patients • NCT04598451

14%

HYPERTENSION

12%

INSOMNIA

12%

COVID-19

BLOOD LACTATE DEHYDROGENASE INCREASED

HYPERTRIGLYCERIDAEMIA

MYOPATHY

HEADACHE

DEPRESSION

DERMATITIS ACNEIFORM

INJECTION SITE ERYTHEMA

ORAL CANDIDIASIS

NASOPHARYNGITIS

URINARY TRACT INFECTION

LEUKOCYTURIA

INCREASED TENDENCY TO BRUISE

SPINAL COMPRESSION FRACTURE

GLOMERULONEPHRITIS

DRUG ERUPTION

SKIN INFECTION

HYPOPROTEINAEMIA

RESPIRATORY FAILURE

TACHYCARDIA

VISION BLURRED

WEIGHT INCREASED

PEMPHIGUS

ERYSIPELAS

VOMITING

COVID-19 PNEUMONIA

GASTROENTERITIS

PNEUMONIA

DIABETES MELLITUS

100%

80%

60%

40%

20%

Study treatment Arm

Efgartigimod PH20 SC

Placebo PH20 SC

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: efgartigimod PH20 SCExperimental Treatment1 Intervention

Patients receiving efgartigimod PH20 SC treatment

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

efgartigimod PH20 SC

2021

Completed Phase 3

~490

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 (ITP) include FcRn antagonists like Efgartigimod PH20, thrombopoietin receptor agonists (TPO-RAs) such as romiplostim and eltrombopag, and immunosuppressants like corticosteroids. FcRn antagonists work by reducing the levels of pathogenic IgG antibodies that contribute to platelet destruction, thereby increasing platelet counts. TPO-RAs stimulate the production of platelets by activating the thrombopoietin receptor on megakaryocytes. Immunosuppressants reduce the immune system's attack on platelets. These treatments are crucial for ITP patients as they help manage symptoms, reduce bleeding risks, and improve quality of life by increasing platelet counts.