Searchable abstracts of presentations at key conferences in endocrinology

ea0035p160 | Calcium and Vitamin D metabolism | ECE2014

PARADIGHM: a natural history registry for patients with chronic hypoparathyroidism

Bowlin Steven , Sadowski Tara , Vokes Tamara , Bilezikian John , Clarke Bart , Mannstadt Michael , Shoback Dolores , Lagast Hjalmar

Chronic hypoparathyroidism affects ~2.5/10 000 individuals in the European Union and is classified as a rare disorder. There is limited knowledge of the natural history and epidemiology of this disease. Publications frequently represent a single center’s experience and information across the population of patients with hypoparathyroidism is lacking and needed. To fill the gap, a voluntary global hypoparathyroidism patient registry (PARADIGHM) was initiated by NPS Pharmace...

ea0073oc6.5 | Oral Communications 6: Calcium and Bone | ECE2021

Safety, tolerability and pharmacodynamics of AZP-3601, a novel long-acting PTH analog, in healthy adults: Data from a randomized, double-blind, placebo-controlled phase 1 study

Allas Soraya , Ovize Michel , Culler Michael D. , Geraul Clarisse , Jeroen van de Wetering , Mannstadt Michael

Hypoparathyroidism is a rare disease characterized by a deficiency in parathyroid hormone (PTH) that results in hypocalcemia and hyperphosphatemia. Current treatment approaches, including high dose oral calcium and active vitamin D, as well as recombinant human PTH (1–84), do not provide adequate or consistent control of either serum calcium or clinical symptoms over a full 24-hour period. AZP-3601 is a novel 36 amino-acid PTH analog that has been designed to potently bi...

ea0099p446 | Calcium and Bone | ECE2024

Investigating the etiology of non-surgical hypoparathyroidism: insights from a sponsored genetic testing program

Mannstadt Michael , Marelli Claudio , Sridhar Ananth , Smith Lyndsay , Roberts Mary Scott , Adler Scott , Mathew Arun

Hypoparathyroidism is a rare endocrine disorder characterized by inadequate production of parathyroid hormone to maintain normal blood calcium levels. Hypoparathyroidism is most frequently caused by damage to or removal of the parathyroid glands but can also be associated with genetic etiologies. Genetic forms of hypoparathyroidism can present as isolated or as part of a syndrome and include disorders of parathyroid gland formation, parathyroid hormone secretion, and damage to...

ea0035oc6.5 | Bone, calcium & vitamin D | ECE2014

The effect of recombinant human parathyroid hormone, rhPTH(1–84), on vitamin D metabolism and phosphate homeostasis: Results from phase III 24-Week REPLACE and phase I clinical studies

Brandi Maria Luisa , Beckers Albert , Vokes Tamara , Mannstadt Michael , Bilezikian John , Clarke Bart , Lagast Hjalmar , Shoback Dolores

PTH promotes conversion of 25-hydroxyvitamin D (25[OH]D) to 1,25-dihydroxyvitamin D (1,25[OH]2D), thus stimulating intestinal calcium and phosphate absorption. Because of low PTH levels in hypoparathyroidism, patients are prescribed calcitriol. Patients are predisposed to hyperphosphatemia owing to loss of PTH-stimulated phosphate excretion by the kidneys. Effects of rhPTH(1–84) on vitamin D metabolism and serum phosphate were studied.In ...

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...

ea0049ep291 | Calcium & Vitamin D metabolism | ECE2017

Chronic hypoparathyroidism disease profile from 492 patients in the PARADIGHM™ natural history global registry

Clarke Bart L , Ing Steven , Khan Aliya , Mannstadt Michael , McDermott Michael , Piccolo Rebecca , Shanik Michael H. , Vokes Tamara J , Germak John

PARADIGHM™ is a global registry (NCT01922440) of patients diagnosed with hypoparathyroidism (HPT) ≥6 months regardless of aetiology and management. Routine medical care data were entered using electronic case report forms; the 36-item Short Form Health Survey was completed by patients. Baseline-recorded data are reported for 492 patients enrolled as of 1 December 2016 from 41 centres. At baseline, 78% were women, mean (S.D.) age was 49 (17)...

ea0041ep129b | Bone & Osteoporosis | ECE2016

Similarities in postsurgical vs nonsurgical patients with hypoparathyroidism: post hoc analysis from recombinant human parathyroid hormone (rhPTH[1-84], parathyroid hormone rDNA) REPLACE study

Brandi Maria Luisa , Bilezikian John P , Clarke Bart L , Fraser William , Krasner Alan , Lagast Hjalmar , Li Benjamin , Mannstadt Michael , Rejnmark Lars , Shoback Dolores M , Vokes Tamara J

Hypoparathyroidism, a rare disorder characterized by absent or low levels of parathyroid hormone (PTH), often results from thyroid surgery. However, nonsurgical etiologies are present in >10% of patients. Data about this group of patients are limited.In this post hoc REPLACE (NCT00732615, EudraCT2008-005063-34) analysis, baseline characteristics and response to 50–100 μg/day rhPTH(1-84) in patients with postsurgical or nonsurgical ...

ea0037oc3.5 | Calcium, vitamin D and bone | ECE2015

Three-year safety and efficacy data for recombinant human parathyroid hormone, rhPTH(1-84), in the treatment of adults with hypoparathyroidism: the RACE study

Clarke Bart L , Mannstadt Michael , Vokes Tamara J , Rothman Jeffrey , Warren Mark L , Denham Douglas S , Levine Michael A , Lagast Hjalmar , Bilezikian John P , Shoback Dolores M

Hypoparathyroidism is a rare endocrine deficiency due to inadequate amounts of parathyroid hormone (PTH) and is characterised by hypocalcaemia and hyperphosphataemia. Current management regimens with large amounts of oral calcium and active vitamin D do not adequately control mineral homeostasis and can lead to complications in many patients. The phase III REPLACE and RELAY clinical trials established the efficacy and safety of once-daily s.c. injection of r...

ea0073aep644 | Thyroid | ECE2021

Assessment of clinical burden and practice patterns in patients with chronic hypoparathyroidism in the united states (us): a claims data analysis using diagnosis-based criteria

Sharma Dolly , L Deering Kathleen , Allas Soraya , Culler Michael D. , Loustau Patrick , Weiss Blandine , Michelle Mitchell Deborah , Astolfi Danette , Mannstadt Michael

ObjectivesThere is a paucity of real-world studies analyzing comorbidities, lab testing and treatment patterns of patients with chronic hypoparathyroidism (cHP). This study describes a large cohort of cHP patients identified using a diagnosis-based approach from a US claims database.MethodsThis retrospective study was conducted using a large (130 million individuals) claims database (HealthVerity Closed Payer...

ea0073ep226 | Thyroid | ECE2021

Assessment of clinical burden and practice patterns in patients with chronic hypoparathyroidism in the United States (US): A claims data analysis using a surgery-based approach

Sharma Dolly , L Deering Kathleen , Loustau Patrick , Michael D. Culler , Allas Soraya , Weiss Blandine , Michelle Mitchell Deborah , Astolfi Danette , Mannstadt Michael

ObjectivesThere is a paucity of real-world studies on the clinical burden and practice patterns associated with chronic hypoparathyroidism (cHP). This study assesses the comorbidities, treatment and lab testing patterns in cHP patients identified using surgery-based criteria.MethodsThis was a retrospective study conducted using a large (130 million individuals) US claims database (HealthVerity Closed Payer Cl...