KURZPROTOKOLL Volasertib 1230.28 Öffentlicher Titel Phase I Studie zu Volasertib zusätzlich zur Standard-Salvagetherapie bei Kindern mit AML nach fehlgeschlagener Erstlinienbehandlung Wissenschaftl. Titel Open-label, dose-escalating trial to evaluate the tolerability, toxicity, safety, pharmacokinetics, pharmacodynamics and activity of volasertib added to the standard intensive salvage chemotherapy regimen with liposomal daunorubicine, fludarabine and cytarabine (DNX-FLA) followed by fludarabine and cytarabine (FLA) in children from 3 months to less than 18 years of age with acute myeloid leukaemia after failure of the front-line therapy Kurztitel Volasertib 1230.28 Studienart multizentrisch, offen/unverblindet, einarmig Studienphase Phase I Erkrankung PAED: Leukämien & Lymphome: Rezidiviert/refraktär Ziele - Determination of the maximal tolerated dose of volasertib or the recommended volasertib dose for further studies in combination with standard salvage therapy in paediatric patients with AML after failure of the front-line intensive chemotherapy regimen [ Time Frame: 4 weeks ] [ Designated as safety issue: No ] - Anti-leukaemic activity of volasertib in combination with standard salvage therapy [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Event-free survival (EFS) [ Time Frame: up to 5 years ] [ Designated as safety issue: No ] - Overall survival (OS) [ Time Frame: up to 5 years ] [ Designated as safety issue: No ] - Number of patients with clinically relevant lab value changes of calcium (hyperand/or hypocalcaemia) as judged by the investigator and reported as adverse events (Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or higher) [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events) [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Predose concentration of volasertib before administration of second dose [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Area under the concentration-time curve of volasertib [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Terminal half-life of volasertib in plasma [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Maximum concentration of volasertib [ Time Frame: 8 weeks ] [ Designated as safety issue: No ] - Patients 3 months to <18 years of age at the time of informed consent - Patients with AML after failure of the front-line intensive AML therapy - Lansky score at screening >=50 for patients from 3 months to <12 years - Karnofsky score at screening >=50 for patients from 12 to <18 years - Use of highly effective methods of birth-control, if sexually active - Parents/legal guardians and patients have given written informed consent and informed assent suitable for the respective age group - Down syndrome - Acute promyelocytic leukaemia and treatment-related AML - QTc prolongation - LVSF <30% - Cardiac disease and/or dysfunction Einschlusskriterien Ausschlusskriterien © Universitäres Centrum für Tumorerkrankungen (UCT) am Universitätsklinikum Frankfurt Ohne Gewähr für Richtigkeit oder Vollständigkeit, www.uct-frankfurt.de Stand: 24.04.2016; Seite 1 von 2 KURZPROTOKOLL Volasertib 1230.28 - Active uncontrolled infection - HIV infection, acute or chronic hepatitis - Inadequate lab parameters - Impaired renal function - Pregnancy or nursing - Further exclusion criteria apply Alter 3 Monate bis 17 Jahre Status Aktiv Beginn der Rekrutierung 01.03.2016 Fallzahl 42 Prüfzentren Universitätsklinikum Frankfurt Klinik für Kinder- und Jugendmedizin Theodor-Stern-Kai 7 60590 Frankfurt am Main Maja Taubenroth Tel: 0696301 84348 Sponsoren Boehringer Ingelheim Pharmaceuticals Registrierung in anderen Studienregistern ClinicalTrials NCT02722135 EUDRACT 2015-004625-14 © Universitäres Centrum für Tumorerkrankungen (UCT) am Universitätsklinikum Frankfurt Ohne Gewähr für Richtigkeit oder Vollständigkeit, www.uct-frankfurt.de Stand: 24.04.2016; Seite 2 von 2
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