Clinical trial NCT01973244: A Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of Long-acting Growth Hormone (Somapacitan) Compared to Daily Dosing of Norditropin® SimpleXx® in Children With Growth Hormone Deficiency
Official title: A Randomised, Open-labelled, Active-controlled, Multinational, Dose-escalation Trial Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of a Single Dose of Long-acting Growth Hormone (NNC0195-0092, Somapacitan) Compared to Daily Dosing of Norditropin® SimpleXx® in Children With Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Growth Hormone Disorder
Growth Hormone Deficiency in Children
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2013-10-31
Enrollment: 32
Sex: ALL
Minimum age (years): 6
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Europe and Asia. The aim of the trial is to investigate safety, tolerability, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of a single dose of long-acting growth hormone (NNC0195-0092, somapacitan) compared to daily dosing of Norditropin® SimpleXx® (somatropin) in children with growth hormone deficiency.


Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of GHD (growth hormone deficiency) as defined by two different GH (growth hormone) stimulation tests, peak GH level below or equal to 7.0 ng/ml. For children with three or more pituitary hormone deficiencies only one GH stimulation test will be needed. If in accordance with country specific practice, growth hormone deficiency can be defined by only one GH stimulation test, peak GH level below or equal to 7.0 ng/ml.
* Pre-pubertal children at screening. Boys: Tanner stage 1 and age above or equal to 6 years and below 13 years. Girls: Tanner stage 1 and age above or equal to 6 years and below 12 years
* Body weight above or equal to 16.0 kg and below or equal to 50.0 kg
* Stable GH replacement treatment for at least 3 months

Exclusion Criteria:

* History or presence of malignancy
* Overt diabetes mellitus (fasting blood glucose above or equal to 7.0 mmol/l)

Primary outcomes:
measure: Incidence of adverse events (AEs)
timeFrame: From first administration of trial product and up until day 35 (final visit)
Secondary outcomes:
measure: The area under the insulin-like growth factor I (IGF-I) concentration-time curve
timeFrame: From 0 to 168 hours after dosing
Other outcomes:
Locations:
city: Graz
country: Austria
facility: Novo Nordisk Investigational Site
city: Linz
country: Austria
facility: Novo Nordisk Investigational Site
city: Wien
country: Austria
facility: Novo Nordisk Investigational Site
city: Brussel
country: Belgium
facility: Novo Nordisk Investigational Site
city: Bruxelles
country: Belgium
facility: Novo Nordisk Investigational Site
city: Paris
country: France
facility: Novo Nordisk Investigational Site
city: Toulouse cedex 9
country: France
facility: Novo Nordisk Investigational Site
city: Haifa
country: Israel
facility: Novo Nordisk Investigational Site
city: Jerusalem
country: Israel
facility: Novo Nordisk Investigational Site
city: Kfar Saba
country: Israel
facility: Novo Nordisk Investigational Site
city: Petah Tikva
country: Israel
facility: Novo Nordisk Investigational Site
city: Tel Hashomer
country: Israel
facility: Novo Nordisk Investigational Site
city: Skopje
country: North Macedonia
facility: Novo Nordisk Investigational Site
city: Bergen
country: Norway
facility: Novo Nordisk Investigational Site
city: Ljubljana
country: Slovenia
facility: Novo Nordisk Investigational Site
city: Barcelona
country: Spain
facility: Novo Nordisk Investigational Site
city: Esplugues Llobregat(Barcelona)
country: Spain
facility: Novo Nordisk Investigational Site
city: Santiago de Compostela
country: Spain
facility: Novo Nordisk Investigational Site
city: Vitoria
country: Spain
facility: Novo Nordisk Investigational Site
city: Stockholm
country: Sweden
facility: Novo Nordisk Investigational Site
city: Basel
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Genève
country: Switzerland
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Juul Kildemoes R, Hojby Rasmussen M, Agerso H, Overgaard RV. Optimal Monitoring of Weekly IGF-I Levels During Growth Hormone Therapy With Once-Weekly Somapacitan. J Clin Endocrinol Metab. 2021 Jan 23;106(2):567-576. doi: 10.1210/clinem/dgaa775.
citation: Papathanasiou T, Agerso H, Damholt BB, Hojby Rasmussen M, Kildemoes RJ. Population Pharmacokinetics and Pharmacodynamics of Once-Daily Growth Hormone Norditropin(R) in Children and Adults. Clin Pharmacokinet. 2021 Sep;60(9):1217-1226. doi: 10.1007/s40262-021-01011-3. Epub 2021 Apr 17.
citation: Juul RV, Rasmussen MH, Agerso H, Overgaard RV. Pharmacokinetics and Pharmacodynamics of Once-Weekly Somapacitan in Children and Adults: Supporting Dosing Rationales with a Model-Based Analysis of Three Phase I Trials. Clin Pharmacokinet. 2019 Jan;58(1):63-75. doi: 10.1007/s40262-018-0662-5.
citation: Battelino T, Rasmussen MH, De Schepper J, Zuckerman-Levin N, Gucev Z, Savendahl L; NN8640-4042 Study Group. Somapacitan, a once-weekly reversible albumin-binding GH derivative, in children with GH deficiency: A randomized dose-escalation trial. Clin Endocrinol (Oxf). 2017 Oct;87(4):350-358. doi: 10.1111/cen.13409. Epub 2017 Aug 8.