hrp0092p1-363 | GH and IGFs (2) | ESPE2019

Metabolism of Somapacitan, a Long-Acting Growth Hormone Derivative, in Human Subjects

Damholt Birgitte B , Bjelke Mads , Helleberg Hans , Rasmussen Michael H

Background: Somapacitan is a reversible albumin-binding growth hormone (GH) derivative developed for once-weekly administration in patients with GH deficiency (GHD). It consists of a human GH backbone, similar to endogenous human GH, with one amino acid substitution, attached to an albumin binder via a linker chain.Objective: Absorption, metabolism and excretion (AME) of somapacitan were investigated in a Phase 1 trial (...

hrp0092p1-371 | Growth and Syndromes (to include Turner Syndrome) (2) | ESPE2019

Growth Hormone Deficiency (GHD): Assessing Parent Burden for Child Growth Hormone Deficiency Treatment: The Growth Hormone Deficiency - Parent Treatment Burden Measure (GHD-PTB)

Brod Meryl , Rasmussen Michael Højby , Vad Knud , Alolga Suzanne , Bedoin Jacques

Background: Treatment for child GHD requires daily injections, which can be painful and disruptive. For most children, these injections are administered by an adult, usually their parent. Unfortunately, little is known about the burden that a child's treatment places on a parent. The GHD-PTB was developed according to FDA/EMA guidances to address this gap. Items were based on qualitative interviews of 31 parents of children with GHD, ages 4 to <13 year...

hrp0092fc14.5 | GH and IGF5 | ESPE2019

OOnce-weekly Somapacitan vs Daily Growth Hormone (Norditropin®) in Childhood Growth Hormone Deficiency: One-year Results from a Randomised Phase 2 Trial

Sävendahl Lars , Battelino Tadej , Horikawa Reiko , Juul Rasmus Vestergaard , Rasmussen Michael Højby , Saenger Paul

Daily injections are required for human growth hormone (GH) replacement therapy. Somapacitan is a long-acting GH derivative being developed for once-weekly dosing in adults and children using a proven protraction method, currently successfully in use to extend the half-life of insulin and glucagon-like peptide 1.To evaluate the efficacy and safety of three different once-weekly somapacitan doses compared with Norditropin®, a daily GH, ove...

hrp0086rfc8.4 | Growth: Clinical | ESPE2016

A Hybrid Fc-fused Human Growth Hormone, GX-H9, Shows a Potential for Weekly and Semi-monthly Administration in Clinical Studies

Lee EunJig , Schopohl Jochen , Mykola Aryaev , Kim Tae Kyung , Ahn Young-Joo , Woo Jung-Won , Jang Woo Ick , Sung Young-Chul , Keyoung H. Michael

Background, Objective and hypotheses: GX-H9 is a hybrid Fc-based long-acting recombinant human growth hormone (hGH). The safety, tolerability, and PK/PD of single ascending dose in healthy volunteers were assessed to determine GX-H9 doses capable of normalizing IGF-1 level. The PK/PD, safety, efficacy and tolerability of multiple sequential doses of GX-H9 in adult growth hormone deficiency (AGHD) were compared to that of a daily recombinant hGH in AGHD. A Phase 2 study in pedi...

hrp0089fc9.1 | Pituitary, neuroendocrinology and Puberty 1 | ESPE2018

Novel Variants in the POU1F1 Beta Isoform are Associated with Isolated Growth Hormone Deficiency and Combined Pituitary Hormone Deficiency

Hoppmann Julia , Rockstroh-Lippold Denise , Gergics Peter , Nakaguma Marilena , Carvalho Luciani Renata Silveira , Pfaeffle Heike , Jamra Rami Abou , Jorge Alexander , Guo Michael H. , Dauber Andrew , Keller Eberhard , Camper Sally A. , Arnhold Ivo JP , Pfaeffle Roland

Background: Hypopituitarism is characterized by deficiency of one or more anterior pituitary hormones. POU1F1 mutations are the second most frequent known cause of combined pituitary hormone deficiency (CPHD). Patients are typically deficient in GH, TSH, and prolactin, although two unrelated cases were reported with isolated GH deficiency (IGHD). To date, all POU1F1 mutations have been reported for the predominantly expressed alpha isoform, which is a transcr...