Searchable abstracts of presentations at key conferences in endocrinology

ea0049gp66 | Developmental & Protein Endocrinology | ECE2017

Achievement of a suitable basis of comparison in phase 2 and 3 clinical trials (VERTICAL/VISTA, and VELOCITY) comparing somavaratan vs daily rhGH for pediatric GH deficiency

Backeljauw Philippe , Miller Bradley S , Wright Nancy , Maniatis Aristides , Stalvey Michael , Humphriss Eric , Charlton R William , Bright George M

Efficacy of rhGH for treatment of pediatric GH deficiency (PGHD) may depend on several variables at treatment initiation (age, body weight, height velocity (HV), IGF-I SDS, skeletal maturation, GHmax), but may be compromised by poor adherence to required daily injections. Somavaratan is a novel, long-acting rhGH fusion protein that demonstrated clinically meaningful improvements in HV and IGF-I concentration in PGHD patients. Over 200 pre-pubertal GHD subjects have enrolled in...

ea0049gp65 | Developmental & Protein Endocrinology | ECE2017

3-year safety and efficacy Update of the VERTICAL & VISTA trials of somavaratan (VRS-317), a long-acting rhGH, in children with Growth Hormone Deficiency (GHD)

Miller Bradley S. , Moore Wayne V. , Fechner Patricia Y. , Van Meter Quentin L. , Fuqua John S. , Ng David , Humphriss Eric , Charlton R. William , Bright George M.

Requirement for daily rhGH injections is a treatment burden that can compromise adherence and efficacy in patients with GHD. Somavaratan is a novel long-acting rhGH fusion protein in clinical development for pediatric and adult GHD. In a multicenter, randomized Phase 1b/2a study, somavaratan significantly improved height velocity (HV) and IGF-I in pre-pubertal children with GHD. Preliminary efficacy and safety in subjects who have completed 3 years of somavaratan treatment are...

ea0041gp36 | Bone & Calcium Homeostasis | ECE2016

Fontan palliation in children is associated with bone deficits

Petryk Anna , Polgreen Lynda E , Brown Roland , Marino Bradley S , Gremmels David , Shepard Charles , Kelly Aaron S , Miller Bradley S , Rudser Kyle , Kochilas Lazaros K

Background: Survivors with Fontan circulation suffer from chronic systemic hypoperfusion resulting in end-organ injury. Little is known about the effects of these hemodynamic perturbations on bone. We hypothesized that chronic Fontan circulation (>5 years after surgery) would be associated with bone deficits.Methods: Peripheral quantitative computed tomography (pQCT) was performed on 10 Fontan patients (seven males, 11.8±1.7 years) and 11 health...