Abstract Search

ea0090p702 | Pituitary and Neuroendocrinology | ECE2023

A Low Incidence of Transient Anti-Drug Antibodies Is Observed Upon Long-Term Exposure to Lonapegsomatropin in Children With Growth Hormone Deficiency

Holse Mygind Per , Komirenko Allison , Mao Meng , Shu Aimee , Breinholt Vibeke M.

Objectives: Lonapegsomatropin (SKYTROFA; TransCon hGH), a prodrug of somatropin, is approved by the EMA and FDA for once-weekly treatment of pediatric growth hormone deficiency (GHD). Lonapegsomatropin uses TransCon® technology, which combines a parent drug transiently linked to an inert carrier to achieve sustained release. The characterization of anti-drug antibodies (ADAs) was assessed in 3 phase 3 trials of lonapegsomatropin in pediatric GHD.Met...

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ea0090p432 | Pituitary and Neuroendocrinology | ECE2023

Design of the foresiGHt Trial: A Multicenter, Randomized, Placebo- and Active-Controlled Trial to Compare Once-Weekly Lonapegsomatropin to Placebo and Daily Somatropin in Adults with Growth Hormone Deficiency (GHD)

Gilis-Januszewska Aleksandra , Fleseriu Maria , Otto Jorgensen Jens , CJ Yuen Kevin , Hoybye Charlotte , Mao Meng , Kang Jennifer , Song Wenjie , Komirenko Allison , Shu Aimee , Beckert Michael

Background: Adult GHD results from insufficient growth hormone (GH) secretion from the anterior pituitary gland and may represent either a continuation of childhood-onset GHD or GHD acquired during adulthood. Clinically, adult GHD is associated with central adiposity, decreased lean muscle mass, increased fat mass, decreased bone mineral density, and reduced quality of life. Current standard of care consists of GH replacement via daily injections. Lonapegsomatropin (SKYTROFA; ...

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ea0081rc8.6 | Rapid Communications 8: Calcium and Bone | ECE2022

Health related quality of life in adults with hypoparathyroidism in an exploratory analysis of the phase 2 PaTH forward trial of TransCon PTH

Palermo Andrea , Vokes Tamara J , Khan Aliya , Rubin Mishaela , Schwarz Peter , Clarke Bart L , Eriksen Erik , Marcocci Claudio , Pagotto Uberto , Tsourdi Elena , Sikjaer Tanja , Brod Meryl , Mcleod Lori , Markova Denka , Noori Wahidullah , Eng Walter Frank , Shu Aimee , Smith Alden

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...

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

TransCon PTH as a potential hormone replacement therapy for patients with hypoparathyroidism: PaTH forward open-label extension trial week 26 results

Schwarz Peter , Rejnmark Lars , Rubin Mishaela , Vokes Tamara J. , Clarke Bart L. , Ahmed Intekhab , Palermo Andrea , Marcocci Claudio , Pagotto Cristina , Eriksen Erik , Mourya Sanchita , Markova Denka , Pihl Susanne , Shu Aimee , Beckert Michael , Khan Aliya

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...

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ea0090p29 | Calcium and Bone | ECE2023

TransCon PTH Improves Health-Related Quality of Life and Reduces Work Limitations in Adults With Hypoparathyroidism: Patient-Reported Outcomes in the Phase 3 PaTHway Trial

Palermo Andrea , Khan Aliya , Rubin Mishaela , Schwarz Peter , Shoback Dolores M. , Gagnon Claudia , Cetani Filomena , Clarke Bart L. , Tsourdi Elena , Kohlmeier Lynn , Sikjaer Tanja , M Kaiser Stephanie , Lai Bryant , Le John , Ukena Jenny , Sibley Christopher , Shu Aimee , An Xubei , Noori Wahidullah , Smith Alden , Vokes Tamara J.

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...

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Endocrine Abstracts

A Low Incidence of Transient Anti-Drug Antibodies Is Observed Upon Long-Term Exposure to Lonapegsomatropin in Children With Growth Hormone Deficiency

Design of the foresiGHt Trial: A Multicenter, Randomized, Placebo- and Active-Controlled Trial to Compare Once-Weekly Lonapegsomatropin to Placebo and Daily Somatropin in Adults with Growth Hormone Deficiency (GHD)

Health related quality of life in adults with hypoparathyroidism in an exploratory analysis of the phase 2 PaTH forward trial of TransCon PTH

TransCon PTH as a potential hormone replacement therapy for patients with hypoparathyroidism: PaTH forward open-label extension trial week 26 results

TransCon PTH Improves Health-Related Quality of Life and Reduces Work Limitations in Adults With Hypoparathyroidism: Patient-Reported Outcomes in the Phase 3 PaTHway Trial

BiosciAbstracts