E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036476 |
E.1.2 | Term | Prader-Willi syndrome |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess effects of GH-treatment versus placebo in young adults with Prader-Willi Syndrome after reaching final height on - body composition - carbohydrate metabolism - psychosocial functioning - sleep-related breathing disorders - circulating lipids - blood pressure
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E.2.2 | Secondary objectives of the trial |
- To study the effects of GH-treatment versus placebo in young adults with Prader-Willi Syndrome after reaching final height on thyroid hormone levels, IGF-I and IGF binding proteins, adiponectin, ghrelin. - To study compliance to the diet.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Genetically confirmed diagnosis of PWS and -treated with GH during childhood for at least 2 years and -final height is reached or epiphysial fusion is complete and -aged 18-24 |
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E.4 | Principal exclusion criteria |
-non cooperative behaviour -extremely low dietary intake of less than minimal required intake according to WHO -medication to reduce weight (fat)
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E.5 End points |
E.5.1 | Primary end point(s) |
The aim of this double-blind cross-over, placebo-controlled study is to evaluate effects of GH-treatment versus withdrawal of GH, after final height is reached, on weight, body composition (as measured with DXA), psychosocial functioning (as measured with GIT, behavioural questionnaires), carbohydrate metabolism (as measured with OGTT), circulating lipids (total cholesterol, HDL, LDL, Lipoprotein A, TG), and sleep-related breathing (polysomnography) during transition period until the age of 24 years. |
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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 | 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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
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 | Yes |
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 | 5 |
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 | 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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1-10-2011 (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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
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 |