ESPE Abstracts

ESPE Abstracts

Published by BioScientifica

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hrp0098fc4.4 | Adrenals and HPA Axis 1 | ESPE2024

Crinecerfont, a Corticotropin-Releasing Factor Type 1 Receptor (CRF1) Antagonist, Reduced Excess Adrenal Androgens and Glucocorticoid Doses in Children and Adolescents with Classic Congenital Adrenal Hyperplasia: Results from CAHtalystTM Pediatric

Sarafoglou Kyriakie , S. Kim Mimi , Lodish Maya , I. Felner Eric , Martinerie Laetitia , J. Nokoff Natalie , Clemente Maria , Y. Fechner Patricia , G. Vogiatzi Maria , W. Speiser Phyllis , B.G. Rosales Gelliza , Roberts Eiry , S. Jeha George , Farber Robert , L. Chan Jean , Ottosson Lars , Baroncelli Marta , Dou Zelong , Nilsson Ola

Introduction: Children with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (CAH) require glucocorticoid (GC) therapy to replace cortisol insufficiency and reduce excess adrenal androgens. Supraphysiological GC doses are typically required, predisposing patients to GC-related comorbidities. In Phase 2 studies, participants with CAH who received crinecerfont, a novel oral CRF1 antagonist, experienced reduction of the adrenal a...
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hrp0098fc6.4 | Fat, Metabolism and Obesity 1 | ESPE2024

Relaxation of Food Control Parameters Based on Improvements in the Food Safe Zone Questionnaire Occurs with Reduction of Hyperphagia in Clinical Trials of Diazoxide Choline Extended Release (DCCR) in Participants with Prader-Willi Syndrome

Bridges Nicola , Gevers Evelien , Yanovski Jack , Salehi Parisa , Hall Shoemaker Ashley , Abuzzahab Jennifer , Obrynba Kathryn , Fleischman Amy , Stevenson David , Wilding John , Mathew Verghese , Viskochil David , Felner Eric , E. McCandless Shawn , Bird Lynne , Lah Melissa , Littlejohn Elizabeth , Shea Heidi , Holland Anthony , Goldstone Anthony , Barrett Timothy , Konczal Laura , Dharmaraj Poonam , Das Urmi , Angulo Moris , Kimonis Virginia , Guftar Shaikh Mohomad , Mejia-Corletto Jorge , Yen Kristen , Gandhi Raj , Ballal Shaila , Hirano Patricia , Cowen Neil , Bhatnagar Anish , Huang Michael , Butler Merlin , Miller Jennifer

Background: Prader-Willi syndrome (PWS) is a rare genetic neurobehavioral-metabolic disorder characterized by hyperphagia and behavioral/psychological complications. With no approved therapies to treat hyperphagia, disease management requires strict dietary and environmental controls to restrict access to food. DCCR is an oral, once-daily medication currently under development for the treatment of PWS.Objective: The obje...
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hrp0098fc11.4 | Fat, Metabolism and Obesity 2 | ESPE2024

Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome from the Completed C601 (DESTINY PWS) and C602 Open Label Extension (OLE) Studies

Gevers Evelien , Bridges Nicola , Yanovski Jack , Felner Eric , Salehi Parisa , Hall Shoemaker Ashley , Fleischman Amy , Goldstone Anthony , Angulo Moris , Stevenson David , Obrynba Kathryn , Guftar Shaikh M , Holland Anthony , Mathew Verghese , Viskochil David , E. McCandless Shawn , Bird Lynne , Lah Melissa , Kimonis Virginia , Abuzzahab Jennifer , Konczal Laura , Littlejohn Elizabeth , Shea Heidi , Dharmaraj Poonam , Das Urmi , Mejia-Corletto Jorge , Wilding John , Harwood Katerina , Yen Kristen , Gandhi Raj , Gong Jing , Hirano Patricia , Cowen Neil , Bhatnagar Anish , Huang Michael , Butler Merlin , Miller Jennifer

Background: Prader-Willi syndrome (PWS) is a rare genetic neurobehavioral-metabolic disorder characterized by hyperphagia and behavioral/psychological complications. No approved therapies exist for treating hyperphagia in patients with PWS. DCCR is an oral, once-daily medication currently under development for the treatment of PWS.Objective: The objective was to determine the efficacy of investigational DCCR on hyperphag...
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