hrp0082p2-d3-616 | Turner Syndrome | ESPE2014

Improvements in Bone Mineral Density in Girls of Prepubertal Age with Turner Syndrome

Malievsky Oleg , Volevodz Natalya , Peterkova Valentina

Background: Patients with Turner’s syndrome (TS) develop osteoporosis, resulting from chromosomal deficiency and estrogen deficiency by gonadal dysgenesis.Objective and hypotheses: The aim of this study was to assess bone mineral density (BMD) and parameters of bone remodeling during somatropin therapy in prepubertal girls with TS.Population and/or methods: We examined 22 girls with TS of the age of 11–15 years (the mean ...

hrp0086rfc8.6 | Growth: Clinical | ESPE2016

A Six-Month Safety and Efficacy Study of TransCon hGH Compared to Daily hGH in Pre-Pubertal Children with Growth Hormone Deficiency (GHD)

Chatelain Pierre , Malievsky Oleg , Radziuk Klaudziya , Senatorova Ganna , Beckert Michael

Background: TransCon hGH is a long-acting prodrug of recombinant human growth hormone (hGH) that releases fully active unmodified hGH into the blood compartment. This presentation will detail the final safety and efficacy results of TransCon hGH in a Phase 2 study in children with GHD over a treatment period of six months.Objective and hypotheses: The objective of this Phase 2 study in GHD was to investigate 1) safety and tolerability, 2) pharmacokinetic...

hrp0084p2-407 | GH & IGF | ESPE2015

A Phase 2, 6-Month, Randomised, Active-Controlled, Safety and Efficacy Study of TransCon hGH Compared to Daily Human GH in Children with GH Deficiency

Chatelain Pierre , Malievsky Oleg , Radziuk Klaudziya , Senatorova Ganna , Beckert Michael

Background: TransCon human GH (hGH) is a long-acting prodrug of recombinant hGH that releases fully active unmodified hGH. TransCon hGH was shown in phase 1 studies in healthy volunteers and a phase 2 study in adults with hGH deficiency to: i) be safe and well tolerated, ii) provide dose-dependent hGH levels, with same peak level and exposure compared to daily GH, iii) be suitable for a once-weekly dosing regimen, and iv) induce an IGF1 response within the normal range. This o...

hrp0084fc3.2 | Diabetes | ESPE2015

Experience with Molecular Diagnosis in 48 Cases of Neonatal Diabetes Mellitus Using Targeted Next-Generation Sequencing

Tikhonvich Yulia , Vasilyev Evgeny , Petrov Vasily , Malievsky Oleg , Petryaikina Elena , Ribkina Irina , Stotikova Olga , Tiulpakov Anatoly

Background: Neonatal diabetes mellitus (NDM) comprises a group of monogenic disorders caused by mutations in genes involved in pancreatic development or insulin secretion. Accurate and rapid molecular diagnosis of NDM is pivotal for making decision on the treatment strategy. Next-generation sequencing (NGS) allows simultaneos analysis of several candidate genes, which facilitates the diagnostic procedure in NDM.Objective and hypotheses: To summarise our ...

hrp0092p1-361 | GH and IGFs (2) | ESPE2019

Long-Term Safety of a Once-Weekly Somatrogon (hGH-CTP): 4-Year Results of a Phase 2 Extension Study in Children with Growth Hormone Deficiency

Zielinska Nataliya , Skorodok Yulia , Malievsky Oleg , Iotova Violeta , Rosenfeld Ron G. , Zadik Zvi , Vander Shelly , Pastrak Aleksandra

Background: Once-daily growth hormone (GH) therapy is an effective treatment for children with growth hormone deficiency (GHD), but compliance wanes with ongoing treatment. A once -weekly GH, somatrogon (hGH-CTP), is being developed to reduce the treatment burden of daily dosing for children and caregivers and potentially improve compliance and long-term efficacy. The impact of once-weekly somatrogon on long-term safety, local tolerability and immunogenicity w...

hrp0086rfc8.7 | Growth: Clinical | ESPE2016

Safety and Tolerability of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): 24-month Complete Dataset Results of a Phase 2 Study in Children with Growth Hormone Deficiency

Zelinska Nataliya , Skorodok Julia , Malievsky Oleg , Rosenfeld Ron G. , Zadik Zvi , Koren Ronit , Vander Shelly , Hart Gili , Radziuk Klaudziya

Background: Daily injections are currently required for growth hormone (GH) replacement therapy, which may cause poor compliance, inconvenience and distress for patients. MOD-4023 is a CTP-modified human GH (hGH) developed for once-weekly administration in growth hormone deficient (GHD) adults and children.Objective and hypotheses: In the present Phase 2 study, the safety and tolerability of once-weekly subcutaneous (SC) administration of MOD-4023 were a...

hrp0084p1-114 | Puberty | ESPE2015

Screening of Mutations in Idiopathic Hypogonadotropic Hypogonadism Using a Targeted Next-Generation Sequencing Approach

Kolodkina Anna , Kareva Maria , Kalinchenko Natalia , Raygorodskaya Nadezhda , Malievsky Oleg , Fidelina Olga , Vasilyev Evgeny , Petrov Vasily , Naumova Maria , Tiulpakov Anatoly

Background: To date at least 30 genes are known to be associated with idiopathic hypogonadotropic hypogonadism (IHH). Analysis of all these gene candidates by Sanger sequencing would be expensive, labour-intensive and time-consuming. Recent introduction of next-generation sequencing (NGS) enables simultaneous analysis of multiple gene targets making it an attractive approach in such conditions as IHHObjective and hypotheses: To study the spectrum of mole...

hrp0086rfc8.8 | Growth: Clinical | ESPE2016

Efficacy of Once-Weekly Administration of CTP-Modified Human Growth Hormone (MOD-4023): 24-month Complete Database Results of a Phase 2 Study in Children with Growth Hormone Deficiency

Zelinska Nataliya , Skorodok Julia , Malievsky Oleg , Rosenfeld Ron G. , Zadik Zvi , Koren Ronit , Vander Shelly , Hart Gili , Raduk Dmitri

Background: Growth hormone (GH) replacement therapy currently requires daily injections, which may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) has been developed by OPKO Biologics for once-weekly administration in growth hormone-deficient (GHD) adults and children.Objective and hypotheses: The 24-month efficacy of once-weekly subcutaneous (SC) administration of MOD-4023 was evaluated in a Phase 2 study in G...

hrp0082p1-d3-163 | Growth (2) | ESPE2014

Six Months Follow-Up Pharmacokinetics and Pharmacodynamics Profile of Once-weekly, CTP-modified Human GH (MOD-4023;): phase 2 Dose Finding Study in Children with GHD Deficiency

Hart Gili , Zadik Zvi , Kradziu Klaudziya , Zelinska Nataliya , Malievsky Oleg , Iotova Violeta , Skorodok Julia , Koren Ronit , Amitzi Leanne , Fima Eyal

Background: GH replacement therapy currently requires daily injections, which may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) is being developed for once-weekly administration in GH deficient (GHD) children. In the present study the longer term pharmacokinetics (PK) and pharmacodynamics (PD) profile of MOD-4023 in GHD naïve children was assessed.Objective and hypotheses: To assess the long acting prope...

hrp0084p2-294 | Diabetes | ESPE2015

The Prevalence of Different Subtypes of Maturity-Onset Diabetes of the Young in Russian Federation as Defined by Targeted Next-Generation Sequencing

Zubkova Natalya , Gioeva Olesya , Tichonovich Yulia , Petrov Vasily , Vasilyev Evgeny , Malievsky Oleg , Kiyaev Alexey , Timofeev Alexey , Tiulpakov Anatoly

Background: Among the currently known variants of maturity-onset diabetes of the young (MODY) subtypes 1–3 are the most prevalent, while their relative frequencies vary in different populations. Other types of MODY are more rare, although the studies addressing their prevalences are limited. Recent implementation of next-generation sequencing (NGS) enables simultaneos analysis of multiple candidate genes making it an attracive approach in various monogenic disorders, incl...