NCT03102593

A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP

A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 38 (actual)
• Sponsor: argenx · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.

Detailed description

This is a randomized, double-blind, placebo-controlled Phase II study in which approximately 36 patients will be randomized in a 1:1:1 ratio to receive either ARGX-113 Dose A, or ARGX-113 Dose B body weight or placebo in 4 infusions administered 1-week apart in addition to Standard-of-Care (SoC) treatment. Patients aged 18 to 85 years (inclusive) with confirmed primary immune thrombocytopenia (ITP) who have a platelet count ˂ 30 × 109/L and who are receiving oral corticosteroids and/or permitted oral immunosuppressants and/or Thrombopoietin receptor (TPO-R) agonist as SoC which must be maintained on a stable dose and frequency for at least 4 weeks prior to Screening. The study will include a 2-week Screening, a 3-week Treatment period, and an 21-week follow-up (FU) period. The study is followed by an open label period where patients will be given the option to be treated with ARGX-113 Dose A in cycles of 4 weekly infusions with a minimum of 4 weeks apart. Patients may receive rescue therapy during the study at the discretion the investigator when deemed medically necessary.

Conditions

• Primary Immune Thrombocytopenia

Interventions

Type	Name	Description
DRUG	ARGX-113	ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
OTHER	Placebo	ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly

Timeline

• Start date: 2017-03-13
• Primary completion: 2019-04-09
• Completion: 2019-04-09
• First posted: 2017-04-06
• Last updated: 2023-07-25

Locations

30 sites across 10 countries: Austria, Belgium, Czechia, France, Germany, Hungary, Poland, Spain, Ukraine, United Kingdom