Efgartigimod IV for adolescents (12–17) with chronic immune thrombocytopenia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase 2/3 double-blind randomized study enrolls participants aged 12 to less than 18 with primary chronic ITP lasting more than 12 months and low platelet counts. In the double-blind treatment period participants are randomized 2:1 to receive efgartigimod IV or placebo for up to 24 weeks, after which all participants receive open-label efgartigimod for at least one year and may continue into a second year, followed by an ~8-week off-drug follow-up for a total study duration up to 138 weeks. Eligible participants must have prior ITP therapy (corticosteroids, IVIg, anti-D, TPO‑RAs, or rituximab) with documented prior response to at least one therapy and documented insufficient response to others. The primary goal is to confirm the appropriate pediatric dose and characterize safety and effects on platelet counts compared with placebo while collecting longer-term open-label safety data.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Is aged 12 to less than 18 years when completing the informed consent process
* Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete
* Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.
* Has documented prior response, defined as 1 platelet count of ≥50 × 10\^9/L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin
* Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy
* Has documented mean platelet count of less than 30 x10\^9/L

Exclusion Criteria:

* Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP
* Nonimmune thrombocytopenia
* ITP-associated critical or severe bleeding
* History of hereditary thrombocytopenia

Where it's running

• Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants
  Bordeaux, France Recruiting
• Centre Hospitalier Universitaire de Grenoble - Hopital Couple - Enfant
  La Tronche, France Recruiting
• CHU de Nancy Hopitaux de Brabois
  Vandœuvre-lès-Nancy, France Recruiting
• Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)
  Berlin, Germany Recruiting
• Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik
  Freiburg im Breisgau, Germany Recruiting
• Gaslini Children's Hospital
  Genoa, Italy Recruiting

Study contacts

• Study coordinator: Sabine Coppieters, MD
• Email: clinicaltrials@argenx.com
• Phone: 857-350-4834

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.