Recruiting

Efgartigimod IV for Chronic ITP in Adolescents

What is being tested

Efgartigimod IV

+ Placebo IV

BiologicalOther

Who is being recruted

Cytopenia+15

+ Autoimmune Diseases

+ Blood Coagulation Disorders

From 12 to 17 Years

+10 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Placebo-ControlledPhase 2 & 3

Interventional

Study Start: October 2025

See protocol details

Summary

Principal Sponsorargenx

Study ContactSabine Coppieters, MD

Last updated: February 10, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: October 20, 2025

Actual date on which the first participant was enrolled.

This study aims to determine the correct dosage of a medication called efgartigimod IV for treating young patients, aged 12 to less than 18 years, who have a condition known as chronic immune thrombocytopenia (ITP). Chronic ITP is a disorder where the immune system mistakenly attacks and destroys platelets, which are crucial for blood clotting. Finding the right dose of efgartigimod IV is important because it could help manage this condition more effectively in teenagers, potentially improving their quality of life. Participants in this study will first be randomly assigned to receive either the efgartigimod IV or a placebo through an IV, without knowing which one they are receiving, for up to 24 weeks. After this period, all participants will be given efgartigimod IV in an open-label setting, where both the participants and the researchers know what is being administered, for the first year. They may continue into a second year of treatment if needed. After the treatment phases, there will be an 8-week follow-up period without the study drug to monitor the participants' condition. The entire study lasts up to 138 weeks, and it aims to carefully measure the effects of the drug on platelet levels and overall safety.

Official TitleA 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

NCT07194850

Principal Sponsorargenx

Study ContactSabine Coppieters, MD

Last updated: February 10, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

24 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 12 to 17 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

CytopeniaAutoimmune DiseasesBlood Coagulation DisordersBlood Platelet DisordersHematologic DiseasesHemic and Lymphatic DiseasesHemorrhageHemorrhagic DisordersImmune System DiseasesPathologic ProcessesPurpuraPurpura, ThrombocytopenicSigns and SymptomsSkin ManifestationsPathological Conditions, Signs and SymptomsThrombocytopeniaPurpura, Thrombocytopenic, IdiopathicThrombotic Microangiopathies

Criteria

6 inclusion criteria required to participate

Has documented mean platelet count of less than 30 x10^9/L

Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy

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

Show More Criteria

4 exclusion criteria prevent from participating

ITP-associated critical or severe bleeding

History of hereditary thrombocytopenia

Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP

Nonimmune thrombocytopenia

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

2 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental

Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)

Group II

Placebo

Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 7 locations

Recruiting

Hospital Sant Joan de Deu Barcelona

Esplugues de Llobregat, SpainOpen Hospital Sant Joan de Deu Barcelona in Google Maps

Recruiting

Hospital Infantil Universitario Nino Jesus (HIUNJS)

Madrid, Spain

Recruiting

Hospital Materno-Infantil Universitario Gregorio Maranon

Madrid, Spain

Recruiting

Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)

Cardiff, United Kingdom

Recruiting

7 Study Centers