Endocrine Abstracts

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)...

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 ...

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...

Background: Patients with hypoparathyroidism experience significant physical and cognitive symptoms and reduced health-related quality of life (HRQoL). Conventional therapy for hypoparathyroidism does not fully alleviate diminished HRQoL. The Hypoparathyroidism Patient Experience Scales (HPES) were developed to assess disease-specific physical and cognitive symptoms as well as the impact of hypoparathyroidsm on HRQol. TransCon PTH, an investigational long-acting prodrug of par...

Background: RACE is an open-label study that assessed the long-term safety and efficacy of recombinant human parathyroid hormone 1–84 (rhPTH[1–84]) for the treatment of hypoparathyroidism in adults (ClinicalTrials.gov identifier NCT01297309). Here, we present 6-year safety and efficacy data.Methods: Patients initially received 25 or 50 μg of rhPTH(1–84) subcutaneously, once daily, with stepwise dose adjustments of 25 μg (up or do...

Hypoparathyroidism (HPT) is characterised by hypocalcaemia and impaired renal phosphate excretion and calcium conservation. Oral calcium supplements and calcitriol can improve serum calcium levels but lack the physiologic effects of PTH on renal reabsorption of calcium. RACE is an ongoing open-label study evaluating the long-term safety of recombinant human parathyroid hormone 1-84 (rhPTH[1-84], parathyroid hormone rDNA) in adults with HPT (NCT01297309). In this interim analys...

PARADIGHM™, a global, prospective, observational registry of patients with chronic hypoparathyroidism (HPT), began enrolment in 2013 to collect data on the natural history of chronic HPT (ClinicalTrials.gov NCT01922440). Since initiation, recombinant human PTH, rhPTH(1-84), has been approved in the United States and Europe as an adjunctive treatment for adult patients with HPT. The protocol for the registry (now a European Medicines Agency–designated postm...

Hypoparathyroidism is a disorder of mineral homoeostasis due to parathyroid hormone (PTH) deficiency. Conventional treatment with oral Ca and calcitriol may maintain serum Ca levels but does not replace other physiologic PTH effects. RACE is an ongoing open-label study of recombinant human PTH1-84 (rhPTH1-84) for hypoparathyroidism treatment in adults (ClinicalTrials.gov NCT01297309). Patients initially received rhPTH1-84 25 or 50 μg/day subcutaneously, with 25-μg in...

BackgroundHypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH), resulting in hypocalcemia, hyperphosphatemia, hypercalciuria, and a reduced quality of life (QoL). PTH replacement therapy should restore physiologic levels of PTH and restore downstream physiologic levels of calcitriol, promoting independence from Ca and active vitamin D supplements and normalization of QoL. TransCon PTH is an investigational long-acting p...

Background: Individuals with hypoparathyroidism often experience a range of symptoms associated with reduced health-related quality of life (HRQoL) and work productivity. Conventional therapy aims to alleviate hypocalcemia and acute symptoms but fails to restore normal parathyroid hormone (PTH) physiology or improve HRQoL. In the PaTHway trial, 79% of participants treated with TransCon PTH vs. 5% placebo (P<0.0001) met the primary efficacy endpoint (normal serum c...