NCT06544499

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

A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 69 (estimated)
• Sponsor: argenx · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Primary Immune Thrombocytopenia (ITP)

Interventions

Type	Name	Description
BIOLOGICAL	Efgartigimod IV	Intravenous infusion of efgartigimod
OTHER	Placebo IV	Intravenous infusion of placebo

Timeline

• Start date: 2024-10-18
• Primary completion: 2028-06-01
• Completion: 2028-06-01
• First posted: 2024-08-09
• Last updated: 2026-03-11

Locations

94 sites across 17 countries: United States, Austria, Bulgaria, China, Croatia, Czechia, France, Germany, Hungary, Ireland, Italy, Poland, Portugal, Romania, Serbia, Spain, United Kingdom

Regulatory

• FDA-regulated drug study