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ea0099oc2.6 | Oral Communications 2: Calcium and Bone | Part I | ECE2024

CALIBRATE: A phase 3, randomized, open-label study evaluating the efficacy and safety of encaleret (CLTX-305) compared to standard of care in participants with autosomal dominant hypocalcemia type 1 [NCT05680818]

Rejnmark Lars , Mannstadt Michael , Brandi Maria Luisa , Ozono Keiichi , Tebben Peter , Mathew Arun , Roberts Mary Scott , Adler Scott , Gafni Rachel

Autosomal dominant hypocalcemia type 1 (ADH1), caused by pathogenic gain-of-function calcium-sensing receptor gene (CASR ) variants, is characterized by low parathyroid hormone (PTH) levels, hypocalcemia, hypercalciuria, hyperphosphatemia and hypomagnesemia. Current standard-of-care (SoC) (calcium and active vitamin D) can exacerbate hypercalciuria, which may result in renal complications. Encaleret is an investigational oral calcilytic, functioning as a negative allo...

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ea0081p548 | Calcium and Bone | ECE2022

Disease characteristics, disability, and quality of life in adult HPP patients with muscular symptoms and pain without skeletal manifestations – a cross-sectional analysis from the Global HPP Registry

Dahir Kathryn M. , Angel Martos-Moreno Gabriel , Linglart Agnes , Petryk Anna , Kishnani Priya S. , Rockman-Greenberg Cheryl , Martel Samantha E. , Ozono Keiichi , Hogler Wolfgang , Seefried Lothar

Background: Hypophosphatasia (HPP) is a rare, inherited metabolic disease caused by deficient activity of tissue nonspecific alkaline phosphatase (TNSALP).Methods: Baseline/pretreatment data from the Global HPP Registry were analyzed to compare HPP disease burden between adults (≥18 years of age) with skeletal manifestations (history of rickets, biopsy-proven osteomalacia, recurrent or poorly healing fractures/pseudofractures, etc; Skeletal group) ...

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Endocrine Abstracts

CALIBRATE: A phase 3, randomized, open-label study evaluating the efficacy and safety of encaleret (CLTX-305) compared to standard of care in participants with autosomal dominant hypocalcemia type 1 [NCT05680818]

Disease characteristics, disability, and quality of life in adult HPP patients with muscular symptoms and pain without skeletal manifestations – a cross-sectional analysis from the Global HPP Registry

BiosciAbstracts