Searchable abstracts of presentations at key conferences in endocrinology

ea0099ep23 | Diabetes, Obesity, Metabolism and Nutrition | ECE2024

3-year setmelanotide weight outcomes in patients with bardet-biedl syndrome and obesity

Yanovski Jack , Angel Martos-Moreno Gabriel , Malhotra Sonali , Yuan Guojun , Chung Wendy , Dollfus Helene , Clement Karine

Background: Bardet-Biedl Syndrome (BBS) is a rare genetic disease in which impaired melanocortin-4 receptor (MC4R) signaling leads to hyperphagia and obesity. In an index Phase 3 trial, broad clinical benefit was observed in patients with BBS based on improvement or stabilization in ≥1 measure of weight, hunger, or quality of life with 1 year of treatment with the MC4R agonist setmelanotide. Reported here are long-term extension (LTE) weight outcomes after 3 years of set...

ea0090p331 | Diabetes, Obesity, Metabolism and Nutrition | ECE2023

Exploration of Clinical Improvements Following Setmelanotide in Patients With Bardet-Biedl Syndrome

Haqq Andrea M. , Chung Wendy , Dollfus Helene , Iqbal Anoop , A. Martos-Moreno Gabriel , Poitou Christine , Yanovski Jack A. , Malhotra Sonali , Miller Paul , Yuan Guojun , Forsythe Elizabeth , Clement Karine , Argente Jesus

Background: In patients with Bardet-Biedl syndrome (BBS), signaling impairments in the melanocortin-4 receptor (MC4R) pathway lead to hyperphagia and severe obesity, which negatively impact quality of life (QOL). We evaluated the impact of setmelanotide, an MC4R agonist, on age-appropriate weight-related parameters, hunger, and QOL in a Phase 3 trial of patients with BBS to further characterize clinical benefit in this patient population.Methods: Patient...

ea0081p77 | Diabetes, Obesity, Metabolism and Nutrition | ECE2022

Comparison of hyperphagia and problem behaviors in participants with prader-willi syndrome (PWS) receiving diazoxide choline extended-release (DCCR) with matched participants in PATH for PWS (PfPWS)

Gevers Evelien , Strong Theresa , Miller Jennifer , Felner Eric , Goldstone Tony , Bridges Nicola , Yanovski Jack , Bird Lynne , Butler Merlin , Obrynba Kathryn , Lah Melissa , Shoemaker Ashley , Mejia-Corletto Jorge , Stevenson David , Wilding John , Kimonis Virginia , Abuzzahab Jennifer , Konczal Laura , Mathew Verghese , Cowen Neil , Woloschak Michael , Bhatnagar Anish

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...

ea0081ep360 | Diabetes, Obesity, Metabolism and Nutrition | ECE2022

Long-term safety of diazoxide choline extended-release (DCCR) tablets in patients with prader-willi syndrome

Gevers Evelien , Miller Jennifer , Butler Merlin , Bridges Nicola , Goldstone Tony , Obrynba Kathryn , Salehi Parisa , Felner Eric , Bird Lynne , Shoemaker Ashley , Konczal Laura , Lah Melissa , Yanovski Jack , Angulo Moris , Mejia-Corletto Jorge , Stevenson David , Wilding John , Abuzzahab Jennifer , Shaikh Guftar , Viskochil David , Mathew Verghese , Yen Kristen , Woloschak Michael , Bhatnagar Anish

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...