A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP

Status: Recruiting
Location: See all (3) locations...
Intervention Type: Other, Biological
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY

The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 17
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Romania
Institutul Clinic Fundeni
RECRUITING
Bucharest
Spain
Hospital Infantil Universitario Nino Jesus (HIUNJS)
RECRUITING
Madrid
Hospital Materno-Infantil Universitario Gregorio Maranon
RECRUITING
Madrid
Contact Information
Primary
Sabine Coppieters, MD
clinicaltrials@argenx.com
857-350-4834
Time Frame
Start Date: 2025-10-20
Estimated Completion Date: 2030-10
Participants
Target number of participants: 24
Treatments
Experimental: Efgartigimod IV
Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)
Placebo_comparator: Placebo IV
Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)
Sponsors
Leads: argenx

This content was sourced from clinicaltrials.gov

Similar Clinical Trials