Кафедра педіатрії № 1 та неонатології
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Browsing Кафедра педіатрії № 1 та неонатології by Author "Ammer, Nicola"
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Item Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Macimorelin in Children with Suspected Growth Hormone Deficiency : An Open-Label, Group Comparison, Dose-Escalation Trial(2021) Csаkvаry, Violetta; Ammer, Nicola; Bagci, Ekaterine; Bolshova, Olena; Damholt, Birgitte Bentz; Katanic, Dragan; Mikhailova, Evgenia; Muzsnai, Ágota; Radu, Dmitri; Senatorova, Ganna; Szalecki, Mieczysław; Teifel, Michael; Vajda, Zsolt; Zelinska, Nataliya; Chaychenko, TetyanaDiagnosis of growth hormone deficiency (GHD) in children requires the use of provocative growth hormone (GH) stimulation tests, which can have limited reliability and are potentially contraindicated in some patients. This is the first paediatric study to test the safety, tolerability, and pharmacokinetics (PK)/pharmacodynamics (PD) of macimorelin, an oral GH secretagogue, approved for diagnosis of adult GHD. Methods: In this open-label, group com parison, single-dose escalation trial (EudraCT 2018-00198823), sequential cohorts of patients (C1–C3) received ascending single doses of macimorelin: 0.25 (C1), 0.5 (C2), and 1.0 (C3) mg/kg. Primary endpoints were safety and tolerability, and secondary endpoints were PK/PD. Results: Twenty-four patients aged between 2 and <18 with suspected GHD participated in the study. No macimorelin-related adverse events were reported, and macimorelin was well tolerated.