A Study of Zomacton in Children With Growth Hormone Deficiency

Study Overview
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Brief Summary
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This trial is set up to compare Zomacton to Genotropin for the treatment of growth hormone deficiency in children. The children will be treated for 1 year. Half of the patients will be treated with Genotropin and half with Zomacton. During this time they will be dosed every day by themselves or their parents at home in the evening. There will be 138 patients in the trial from age 3 to age 11. The patients cannot have been treated before with growth hormone and the patients must have a proven growth hormone deficiency, this will be shown by a specific test that will be performed before the trial in the local clinic and once during the trial. During the time of the treatment the patients will come to visit the clinic every 3 months. At these visits their heights will be measured, blood samples will be taken, physical examinations will be performed and questions about their health will be asked. At 2 times in the trial they will have a hand x-ray taken to measure the bone age. At the end of the trial the patients will stop the treatment and continue on one of the marketed products available to treat growth hormone deficiency.

Official Title
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A Randomised, Open-label, Parallel-group, Multi-centre Trial to Compare the Efficacy and Safety for 12 Months of Zomacton to Genotropin in Children With Idiopathic Growth Hormone Deficiency

Conditions
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Growth Hormone Deficiency

Intervention / Treatment
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* Drug: Genotropin
* Drug: Zomacton


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed informed consent Children aged ≤3yrs old and not above 10 yrs for girls or 11 yrs for boys Idiopathic growth hormone deficiency confirmed during the pre-screening period by a standard GH stimulation test (defined as peak level of <10ng/ml pr lower if so required by the country specific board(s) Height SDS <-2 SD of ref value for CA Height velocity SDSCA ≤ 0 SD of ref value for at lease 6 months prior to pre-screening Height recorded for at least 6 months but not more than 18 months of pre-screening The difference between CA-BA≥ 1 A positive locally performed GH stimulation test (defined as a peak plasma level of<9ng/ml or lower if so required by the country specific board(s)) prior to the pre-screening Exclusion Criteria: BA above 9 yrs for girls and 10 yrs for boys Puberty Tanner stage >1 Weight <12 Kg at screening Any prior treatment with GH Closed epiphysis Any diagnosed or suspected syndrome (e.g. Silver -Russell, Turner's or seckel syndrome) which possibly could affect growth Any other diagnosed or suspected endocrine or metabolic disorder Any diagnosed or suspected sever chronic disease Clinical signs of dysmorphic features, malformations or mental retardations Growth failure due to other disorders Previous or present use of drugs that could interfere with GH treatment (e.g. steroids) Diagnosed malignant disease Any abnormal CS lab results that requires further investigation Receipt of an investigational drug within the last 28 days preceding screening or longer if considered possible to influence the outcome of the current trial Any knowledge of hypersensitivity to somatropin or any of the excipients of Zomacton or Genotropin

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1<br> | Drug: Genotropin<br> <br> |
| Experimental: 2<br> | Drug: Zomacton<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height Velocity |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Height SDS |  | 12 months | 
| Height velocity SDS |  | 12 months | 
| Change in IGF-1 and IGFBP-3 |  | 12 months | 
| BA (Bone Age) |  | 12 months | 
| Anti-hGH AB |  | 12 months | 
| AE and tolerability |  | 12 months | 
| CS Changes in safety lab, physical examination and vital signs |  | 12 months |