Health TrialsbyHealthPanel

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

ConditionPrimary Immune Thrombocytopenia (ITP)
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

This trial does not accept healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • Efgartigimod IV

    Biological/Vaccine

    Intravenous infusion of efgartigimod

  • Placebo IV

    Other intervention

    Intravenous infusion of placebo

Treatment groups

69 Participants
are divided into 2 treatment groups
Group A: Efgartigimod IVExperimental treatment 1 intervention
Group B: Placebo IVExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Extent of disease control, defined as the number of cumulative weeks during the 24-week Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L

Time frame
Up to 24 weeks

Secondary outcomes

1

Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP

Time frame
Up to 6 weeks
2

Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP

Time frame
Up to 8 weeks
3

Proportion of participants achieving a platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP

Time frame
Up to 12 weeks
4

Proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions at any time until study week 12 during the DBTP

Time frame
Up to 12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

argenx

Lead sponsor

Source ClinicalTrials.gov
Apply now