E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Zomacton is indicated for the long term treatment of children who have growth failure due to inadequate secretion of growth hormone and for the long-term treatment of growth retardation due to Turner’s Syndrome confirmed by chromosome analysis. |
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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 |
To assess the local tolerability of an individualised dose of Zomacton 10 mg administered by ZomaJet Vision X |
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E.2.2 | Secondary objectives of the trial |
• To assess the frequency of local tolerability reactions when administering an individualised dose of Zomacton 10 mg by ZomaJet Vision X by local assessor • To assess the frequency of local tolerability reactions when administering an individualised dose of Zomacton 10 mg by ZomaJet Vision X by central assessor • To assess the frequency of immediate local reactions after administration of an individualised dose of Zomacton 10 mg by ZomaJet Vision X • To assess the subject’s evaluation of pain associated with transjection of an individualised dose of Zomacton 10 mg administered by ZomaJet Vision X • To assess the subject’s evaluation of itching associated with transjection of an individualised dose of Zomacton 10 mg administered by ZomaJet Vision X
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Subjects between the ages of 3 and 17 years (both inclusive) with growth failure due to inadequate secretion of growth hormone or growth retardation due to Turner’s syndrome. Subjects have for a minimum of 6 months prior to study enrolment been receiving growth hormone therapy. |
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E.4 | Principal exclusion criteria |
• Closed epiphyses • Evidence of progression of an underlying intra-cranial lesion or other active neoplasms (inclusive leukaemia or solid tumours) within 1 year of treatment start • Acute critical illness suffering complications following open-heart surgery, abdominal surgery, multiple accidental trauma, acute respiratory failure, or similar conditions • Any syndromes with an increased risk of chromosomal breakages and malignant diseases (e.g. Down’s syndrome, Bloom syndrome, Fanconi anaemia, neurofibromatosis-1) • Use of any non registered investigational drug within 3 months prior to screening or previous participation in the study • Pregnancy and lactation • Hypersensitivity to any excipients in Zomacton 10 mg and solvents to be used
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E.5 End points |
E.5.1 | Primary end point(s) |
• The number of subjects discontinuing after treatment start due to unacceptable transjection related local tolerability reactions when receiving an individualised dose of Zomacton 10 mg administered by ZomaJet Vision X |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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Definition of the end of trial is 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 | 7 |
E.8.9.1 | In the Member State concerned days | |
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 | 7 |