E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036476 |
E.1.2 | Term | Prader-Willi syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 evaluate the effects of oxytocin on social behaviour in children with Prader-Willi syndrome aged 3-11 years |
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E.2.2 | Secondary objectives of the trial |
- To evaluate the effects on appetite, satiety, food intake and food seeking behaviour in children with PWS aged 3-11 years - To evaluate oxytocin levels in blood and saliva samples before and during intransal oxytocin treatment - To evaluate if there is a difference in effect between once or twice daily adminsitration - To evaluate effects in relation to: BMI and body composition by DXA, IGF-1 and IGF-BP3-levels, genetic subtype |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
In order to be eligible to participate in this study, a subject must meet all of the following criteria: - Genetically confirmed diagnosis of Prader-Willi syndrome - Children aged 3 to 10.99 years - Informed consent - Currently on growth hormone treatment for at least 1 year - Behavioural characteristics such as reduced social reciprocity and interaction, repetitive behaviour or temper tantrums, and/or be in nutritional phase 2b or 3 according to Miller (increased interest in food, hyperphagia) |
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E.4 | Principal exclusion criteria |
A potential subject who meets any of the following criteria will be excluded from participation in this study: - Severe psychiatric problems - Non-cooperative behaviour - Allergic reactions or hypersensitivity for oxytocin - Serious illness - Cardiac abnormalities - Extremely low dietary intake or less than required itnake acoording to WHO - Medication to reduce weight (fat) |
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E.5 End points |
E.5.1 | Primary end point(s) |
Changes in social behaviour assessed by: - Repetitive Behavior Scale-Revised - Social Responsiveness-Scale |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 1 and 3, 4, 7 and 13 months |
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E.5.2 | Secondary end point(s) |
Change in: - Clinical Global Impression scale - Quality of life (DUX25 and DUXPWS) - Social Behaviour (VISK, Oxytocin questionnaire revised) - Hyperphagia (Hyperphagia questionnaire Dykens) - Reading the Mind in the Eyes test, child version - Body Composition (Anthropometric measurements, BMI and DXA-scan) - Social and food related behaviour (diary) - Food intake (diary) - Laboratory parameters (oxytocin in saliva and blood) - Safety parameters (laboratory parameters and medical assessments) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Day 1 and 3, 4, 7 and 13 months |
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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 | Yes |
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) | Yes |
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 | 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.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | No |
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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |