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

Официальное название

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

Обзор

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

Вмешательства

• Биопрепарат Efgartigimod IV
  Intravenous infusion of efgartigimod
• Другое Placebo IV
  Intravenous infusion of placebo

Первичные конечные точки

• Efgartigimod serum concentrations in the DBTP [Срок оценки: Up to 24 weeks]
• Total IgG levels in the DBTP [Срок оценки: Up to 24 weeks]

Вторичные конечные точки (11)

• Efgartigimod serum concentrations over time during the DBTP [Срок оценки: Up to 24 weeks]
• Percent change from baseline in total IgG levels in serum over time during the DBTP [Срок оценки: Up to 24 weeks]
• Incidence of AEs, SAEs and AEs leading to IMP discontinuation [Срок оценки: 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 [Срок оценки: 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 [Срок оценки: Up to 48 weeks]
• Changes from baseline for platelet counts over time [Срок оценки: Up to 76 weeks]
• Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP [Срок оценки: Up to 76 weeks]
• Incidence of ADA and Nab against efgartigimod in serum [Срок оценки: Up to 76 weeks]
• Change from baseline in EQ-5D-5L [Срок оценки: Up to 76 weeks]
• Change from baseline in KIT Child Self-Report and KIT Parent Impact Report [Срок оценки: Up to 76 weeks]
• Change from baseline in peds FACIT-F [Срок оценки: Up to 76 weeks]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Испания · 5 центров

• Hospital Universitari Vall d'Hebron — Barcelona
• Hospital Sant Joan de Deu Barcelona — Esplugues de Llobregat
• Hospital Infantil Universitario Nino Jesus (HIUNJS) — Madrid
• Hospital Materno-Infantil Universitario Gregorio Maranon — Madrid
• Hospital Universitario 12 de Octubre — Madrid

Италия · 4 центра

• Gaslini Children's Hospital — Genoa
• Fondazione IRCCS San Gerardo dei Tintori — Monza
• Bambino Gesu Children's Hospital — Roma
• A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita — Torino

Великобритания · 3 центра

• Cardiff and Vale NHS Trust - University Hospital of Wales (UHW) — Cardiff
• The Royal Hospital for Children - Glasgow Health Board — Glasgow
• Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust — Manchester

Польша · 2 центра

• Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4 — Lodz
• Uniwersytecki Szpital Dzieciecy w Lublinie — Lublin

Сербия · 2 центра

• Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic — Belgrade
• University Clinical Centre of Kragujevac — Kragujevac

Франция · 1 центр

• Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfa — Bordeaux

Германия · 1 центр

• Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) — Berlin

Литва · 1 центр

• Vilnius University Hospital Santaros Klinikos, Children's Hospital — Vilnius

Румыния · 1 центр

• Institutul Clinic Fundeni — Bucharest

Идентификаторы

NCT: NCT07194850 · ARGX-113-2409 · 2025-521055-23-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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