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ea0099p225 | Adrenal and Cardiovascular Endocrinology | ECE2024

Baseline characteristics of children and adolescents with classic congenital adrenal hyperplasia enrolled in CAHtalyst pediatric, a phase 3 Study of Crinecerfont, a corticotropin-releasing factor type 1 receptor antagonist

Sarafoglou Kyriakie , Kim Mimi , Lodish Maya , Felner Eric , Martinerie Laetitia , Nokoff Natalie J. , Clemente Maria , Fechner Patricia Y. , Vogiatzi Maria , Speiser Phyllis , Sturgeon Julia , Roberts Eiry , Jeha George , Chan Jean L. , Farber Robert

Objective: To describe the baseline characteristics of individuals enrolled in CAHtalyst Pediatric (NCT04806451), a randomized, double-blind, placebo-controlled, Phase 3 study evaluating the safety and efficacy of crinecerfont (a corticotropin-releasing factor type 1 receptor [CRF1] antagonist) in children and adolescents with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency (21-OHD CAH).Methods: Key eligibility criteria...

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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...

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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...

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Endocrine Abstracts

Baseline characteristics of children and adolescents with classic congenital adrenal hyperplasia enrolled in CAHtalyst pediatric, a phase 3 Study of Crinecerfont, a corticotropin-releasing factor type 1 receptor antagonist

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)

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

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