Endocrine Abstracts

Objective: To describe real-world safety data on growth hormone therapy (GHT) in paediatric patients with Noonan syndrome (NS) who were enrolled in NordiNet® IOS and the ANSWER®Program.Introduction: Patients with NS have a high prevalence of cardiac defects and an increased risk for leukaemia and certain malignancies compared to the general population. Current safety data do not indicate an association of GHT with worsenin...

Background: Hypothalamic obesity (HO) is an acquired form of severe obesity that can occur following surgical resection of or radiotherapy for brain tumors and is often unresponsive to lifestyle modifications or traditional obesity pharmacotherapies. Here, we report weight- and body composition–related findings from a Phase 2 trial of setmelanotide, a melanocortin-4 receptor agonist, in patients with HO.Methods: A Phase 2, open-label, 16-week trial ...

Background: Hypothalamic injury and impaired melanocortin-4 receptor (MC4R) pathway signaling, often a result of surgery or radiation for a benign tumor, may lead to hypothalamic obesity (HO). After injury, sudden weight gain and appetite changes unresponsive to existing therapies develop. Setmelanotide, an MC4R agonist, is approved for chronic weight management in patients with certain MC4R pathway–associated diseases. We report interim results of a Phase 2 study of setm...

Objective: To report hunger-related results from a Phase 2 trial of setmelanotide in patients with hypothalamic obesity (HO).Methods: A Phase 2, open-label, 16-week trial of setmelanotide in patients aged ≥6 to ≤ 40 years with body mass index (BMI) ≥95th percentile (aged <18 years) or ≥35 kg/m2 (aged ≥18 years) and HO caused by hypothalamic damage secondary to brain tumor, surgical resection, and/or chemothera...

Background: PWS is a rare neurodevelopmental genetic disorder characterized by hyperphagia, obesity, hormonal deficiencies, and problem behaviors for which there are no approved treatment. DCCR administration (100-525 mg/day) up to 52 weeks in participants with PWS improved hyperphagia, behavior, body composition and metabolic markers.Objective: The objective of this study was to compare changes in hyperphagia (using Hyperphagia Questionnaire for Clinica...

Background: Prader-Willi syndrome (PWS), a rare genetic neurobehavioral-metabolic condition, is characterized by hyperphagia, accumulation of excess fat, hypotonia, and behavioral/psychological complications. There are no currently approved medications to treat hyperphagia in patients with PWS; DCCR is under development as a treatment for PWS.Objectives and Methods: The objective was to evaluate long-term safety of DCCR in individuals with PWS. 125 parti...