A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)

Phase 3RecruitingNCT07194850

argenx24 enrolled

Overview

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. More information can be found here: https://clinicaltrials.argenx.com/advancejunior

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Immune Thrombocytopenia (ITP)ITP - Immune Thrombocytopenia ITP

Eligibility

Sex: ALLMin age: 12 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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 Exclusion Criteria: * Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP * Nonimmune thrombocytopenia * ITP-associated critical or severe bleeding * History of hereditary thrombocytopenia

Locations (8)

Gaslini Children's Hospital

Genova, Italy

RECRUITING

Uniwersytecki Szpital Dzieciecy w Lublinie

Lublin, Poland

RECRUITING

Institutul Clinic Fundeni

Bucharest, Romania

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, Spain

RECRUITING

Hospital Sant Joan de Deu Barcelona

Esplugues de Llobregat, Spain

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

Outcomes

Primary Outcomes

Efgartigimod serum concentrations in the DBTP

Time frame: Up to 24 weeks

Total IgG levels in the DBTP

Time frame: Up to 24 weeks

Secondary Outcomes

Efgartigimod serum concentrations over time during the DBTP

Time frame: Up to 24 weeks

Percent change from baseline in total IgG levels in serum over time during the DBTP

Time frame: Up to 24 weeks

Incidence of AEs, SAEs and AEs leading to IMP discontinuation

SAE: Serious adverse event; AE: adverse event

Time frame: Up to 136 weeks

Sustained platelet count response between study weeks 19 and 24 of the DBTP and in OLTP1 for participants receiving placebo in the DBTP

Sustained platelet count defined as achieving platelet counts of ≥50 × 10\^9/L for at least 4 of the 6 study visits

Time frame: Up to 48 weeks

Extent of disease control during the DBTP and during the first 24 weeks of OLTP1 for those participants receiving placebo in the DBTP

Extend of disease defined as the number of cumulative weeks with a platelet count of ≥50 × 10\^9/L

Time frame: Up to 48 weeks

Changes from baseline for platelet counts over time

Time frame: Up to 76 weeks

Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP

The Modified Buchanan and Adix Bleeding Score for pediatric ITP is a semiquantitative assessment tool that measures bleeding signs and symptoms, comprising a score based on a scale of 0 to 5, each representing a different level of severity (0 = no risk; 5 = highest severity).

Time frame: Up to 76 weeks

Incidence of ADA and Nab against efgartigimod in serum

ADA: anti-drug antibodies; Nab: neutralizing antibodies

Time frame: Up to 76 weeks

Change from baseline in EQ-5D-5L

The European Quality of Life 5 Dimensions 5 Level (EQ-5D-5L) is a questionnaire comprised of 5 dimensions. Participants are asked to select the statement in each dimension which best describes their health on the day they complete the questionnaire. Responses in each dimension are coded as a 1-digit number ranging from 1 (no problems) to 5 (extreme problems).

Time frame: Up to 76 weeks

Change from baseline in KIT Child Self-Report and KIT Parent Impact Report

The Kids' ITP Tools (KIT) comprises two disease- specific tools: a self-report form for children aged 12 and older, and a parent impact form. Respondents provide insights into their disease experience using a 1-week recall period. The instrument generates a total score, calculated by summing the items and converting them to a scale of 0 to 100, where higher scores reflect a better disease-specific quality of life (QoL).

Time frame: Up to 76 weeks

Change from baseline in peds FACIT-F

In all participants, the pediatric Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) will use to assess health-related quality of life (HRQoL) before and after treatment. The FACIT-F questionnaire has a score range of 0 to 52, where 0 represents the worst possible fatigue and 52 indicates no fatigue.

Time frame: Up to 76 weeks

A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)

Overview

Conditions

Interventions

Eligibility

Locations (8)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts