E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
children aged 6 years or more receiving growth hormone therapy for the following approved indications (according to Norditropine SPC) -GHD -Turner Syndrome -small for gestational age -chronic renal insufficiency |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to investigate how easy/difficult patients/parents assess growth hormone injection using Norditropin NordiFlex® by quantifying the proportion of patients that find growth hormone injection using Norditropin NordiFlex® easy or very easy as assessed by patient/parent questionnaire at the end of study. |
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E.2.2 | Secondary objectives of the trial |
• Patient/parent overall acceptance/convenience of Norditropin NordiFlex® as assessed by questionnaire at the end of study. • Patients’/parents’ preference to use Norditropin NordiFlex® in comparison to the previous delivery systems. • Influence of NordiFlex PenMate® on overall acceptability/convenience • Compliance to treatment as assessed by patient/parent diary and drug accountability at the end of study • Ease of teaching how to use Norditropin NordiFlex® as assessed by nurse questionnaires at baseline visit. • Evaluation of tolerability, when using Norditropin NordiFlex® by collecting adverse events, serious adverse events, MESI and Clinical Technical Complaints.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Only children whom Norditropin® treatment is indicated in accordance with labelling will be included in this study • Children receiving growth hormone therapy for at least one year • Age ≥ 6 years written informed consent
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E.4 | Principal exclusion criteria |
• Contraindications to Norditropin® growth hormone therapy as stated in the summary of product characteristics. • Patients who have previously been enrolled in this study. • The receipt of any investigational medicinal product within 3 months prior to this trial. • Suffer from a life threatening disease |
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E.5 End points |
E.5.1 | Primary end point(s) |
is to investigate how easy/difficult patients/parents assess growth hormone injection using Norditropin NordiFlex® by quantifying the proportion of patients that find growth hormone injection using Norditropin NordiFlex® easy or very easy as assessed by patient/parent questionnaire at the end of study. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 40 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |