A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
Sponsorargenx

About this trial

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). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week OLTP2. After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks.

More information can be found here: https://clinicaltrials.argenx.com/advancenext

Eligibility criteria

Qualifiers

Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF).

Has documented baseline mean platelet count of <30 x 10^9/L before randomization

Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12 months on the date of informed consent form (ICF) signature.

Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.

Disqualifiers

Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, confound the results of the study or put the participant at undue risk.

Secondary ITP

Nonimmune thrombocytopenia

Autoimmune hemolytic anemia

Trial design

Treatments tested in this trial

  • Efgartigimod IV
  • Placebo IV

Treatment groups

69 Participants
are divided into 2 treatment groups

Sponsors and collaborators