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 (21) 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. More information can be found here: https://clinicaltrials.argenx.com/advancejunior
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
France
Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants
RECRUITING
Bordeaux
Germany
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)
RECRUITING
Berlin
Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik
RECRUITING
Freiburg Im Breisgau
Italy
Gaslini Children's Hospital
RECRUITING
Genoa
Fondazione IRCCS San Gerardo dei Tintori
RECRUITING
Monza
Bambino Gesu Children's Hospital
RECRUITING
Roma
A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita
RECRUITING
Torino
Lithuania
Vilnius University Hospital Santaros Klinikos, Children's Hospital
RECRUITING
Vilnius
Poland
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4
RECRUITING
Lodz
Uniwersytecki Szpital Dzieciecy w Lublinie
RECRUITING
Lublin
Romania
Institutul Clinic Fundeni
RECRUITING
Bucharest
Serbia
Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic
RECRUITING
Belgrade
University Clinical Centre of Kragujevac
RECRUITING
Kragujevac
Spain
Hospital Universitari Vall d'Hebron
RECRUITING
Barcelona
Hospital Sant Joan de Deu Barcelona
RECRUITING
Esplugues De Llobregat
Hospital Infantil Universitario Nino Jesus (HIUNJS)