Paediatric Once Weekly Growth Hormone Study in GH Deficient Children

• Research type

  Research Study

• Full title

  A randomised, open-labelled, single dose, dose-escalation trial investigating safety, tolerability, pharmacokinetics and phamacodynamics of pegylated long-acting human growth hormone (NNC126-0083) compared to Norditropin NordiFlex in growth hormone deficient children

• IRAS ID

  23285

• Eudract number

  2008-008240-25

• ISRCTN Number

  Not Submitted

• Research summary

  This study is a randomised, open-labelled, single dose, dose-escalation trial investigating safety, tolerability, pharmacokinetics and pharmacodynamics of pegylated long-acting human growth hormone (NNC126-0083) compared to Norditropin NordiFlex in growth hormone deficient children.The aim of the project is to develop a long-acting once weekly growth hormone (GH) product which is just as safe and efficacious but has greater convenience and compliance compared to standard once daily GH treatment.The study looks at children who have growth hormone deficiency and therefore must inject themselves with daily doses of replacement growth hormone. As this therapy is initiated at an early age and is life-long it would be advantageous to reduce the number of injections needed.This study will be carried out at either a children??s hospital or within paediatric departments where there are specialised teams with experience and expertise in working with children.Children aged 6-12 years old who are currently on stable growth hormone therapy will be recruited into this trial to investigate the pharmacokinetics (how the drug is absorbed, distributed, metabolised and excreted) and pharmacodynamics (how the drug works in the body) without interference from non-stable treatments.Four different doses of drug will be investigated with 8 children being recruited into each cohort (0.01, 0.02, 0.04 and 0.08 mg protein/kg of study drug). Those children randomised to daily injections will receive 0.035mg/kg/day.Randomisation will be 6:2 - with 6 children receiving long acting GH and 2 receiving daily injections. Treatment allocation is done randomly via a computerised system, however, once allocated both the child and doctor will know which treatment they are on.Children taking part in this study will be on study for 35-53 days depending on the length of time between V1 (screening) and V2 (treatment) which can be between 7-21 days and will consist of 8 hospital visits. Most visits are of a short duration lasting only 1 hour with the exception of V4 which will involve 4 days stay in hospital.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  09/H0720/99

• Date of REC Opinion

  1 Oct 2009

• REC opinion

  Further Information Favourable Opinion