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54th Annual ESPE

Barcelona, Spain
01 Oct 2015 - 03 Oct 2015

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Barcelona, Spain; 1-3 October 2015 Further information

Free Communications

Growth-promoting therapies

hrp0084fc7.1 | Growth-promoting therapies | ESPE2015

Safety and Effectiveness of Increlex® Therapy in Children with Laron Syndrome and Enrolled in the European Increlex® Growth Forum Database in Europe

Bang Peter , Polak Michel , Woelfle Joachim , Houchard Aude , Sert Caroline

Background: The post-authorisation registry, European Increlex® Growth Forum Database (EU-IGFD), initiated in December 2008, collects data in children receiving Increlex (mecasermin (rDNA origin) injection) for growth failure, including Laron syndrome (LS) (severe primary IGF1 deficiency with confirmed GH-receptor mutation).Objective and hypotheses: Report safety and effectiveness data in children with LS.Method: Multicentre, ...

hrp0084fc7.2 | Growth-promoting therapies | ESPE2015

TopiconThermoMatrix™-Mediated Passive Transdermal Delivery of IGF1 Across EpidermFT Full-Thickness Human Skin Equivalent: Towards an Extended-Wear IGF1 Patch

Hsu Stephen , Mangleburg Carl , Yao Hua

Background: There is a need for a convenient and affordable alternative to twice daily s.c. injections for children with IGF1 deficiency (IGFD) and growth failure. We report a novel platform technology, Topicon™ ThermoMatrix™, applied to the transdermal delivery of IGF1 (7649 Da).Objective and hypotheses: We sought to develop a convenient, non-invasive, and affordable transdermal patch formulation capable of achieving passive ...

hrp0084fc7.3 | Growth-promoting therapies | ESPE2015

IGF1 Levels, Complex Formation, and IGF-Bioactivity in GH-Treated Children with Prader–Willi Syndrome

Bakker Nienke , van Doorn Jaap , Renes Judith , Donker Truus , Hokken-Koelega Anita

Background: Children with Prader–Willi syndrome (PWS) attain high serum immunoreactive IGF1 levels during standard dose GH treatment, which leads to concern, but lowering the dose, deteriorates their body composition.Objective and hypotheses: To evaluate serum IGF1, IGFBP3 and acid-labile subunit (ALS) levels, complex formation and IGF-bioactivity in GH-treated PWS children. We hypothesized that GH-treated children with PWS have a normal IGF-bioacti...

hrp0084fc7.4 | Growth-promoting therapies | ESPE2015

A Novel Reversible Albumin-Binding GH Derivative Possesses a Promising Once-Weekly Treatment Profile in Children with GH Deficiency

Zuckerman-Levin Nehama , Gucev Zoran , de Schepper Jean , Rasmussen Michael Hojby , Battelino Tadej , Olsen Minna Braendholt , Savendahl Lars

Background: GH administration restores normal growth in children with GH deficiency (GHD). However, current daily s.c. injection treatment regimens may be inconvenient leading to impaired adherence and subsequently suboptimal treatment outcomes. NNC0195-0092 is a novel, reversible albumin-binding GH developed for once-weekly administration.Objective and hypotheses: This was a randomised, open-label, active-controlled, dose-escalation trial (NCT01973244) ...

hrp0084fc7.5 | Growth-promoting therapies | ESPE2015

12-Month Safety and Efficacy of a Weekly Long-Acting GH (MOD-4023) Compared to Daily Recombinant Human GH Therapy in Pre-Pubertal GH-Deficient Children; Phase 2 Study: Study CP-4-004 Summary

Zadik Zvi , Rosenfeld Ron , Radziuk Klaudziya , Zelinska Nataliya , Malievsky Oleg , Iotova Violeta , Skorodok Julia , Koren Ronit , Amitzi Leanne , Hershkovitz Oren , Hart Gili

Objective and hypotheses: To compare the PK/PD, safety, efficacy, and tolerability of three doses of once-weekly MOD-4023 to that of a daily recombinant human GH (rhGH) formulation in pre-pubertal children with growth failure due to GH deficiency (GHD).Method: The randomised, controlled phase 2 study was conducted in 53 pre-pubertal, hGH-naïve GHD children randomised to receive one of three MOD-4023 doses as a once-weekly s.c. injection (0.25, 0.48,...

hrp0084fc7.6 | Growth-promoting therapies | ESPE2015

Pharmacokinetic and Pharmacodynamics Modelling of MOD-4023 (a Long-Acting Human GH) in GH-Deficient Children

Fisher Dennis , Jaron-Mendelson Michal , Amitzi Leanne , Koren Ronit , Hart Gili

Background, objective, and hypotheses: OPKO Biologics has produced a long-acting human GH (hGH), MOD-4023, containing copies of a naturally-occurring C-terminal peptide (CTP) to markedly increase GH’s in vivo residence. We describe the construction and validation of a pharmacokinetic (PK)/pharmacodynamics (PD) model to characterise the relationship between MOD-4023 dose, MOD-4023 serum concentrations (Cserum), and IGF1 responses in healthy adults, GH-deficient (G...