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 | 17.0 |
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 intranasal oxytocin compared to placebo administration on appetite, satiety and food intake and on social behavior, BMI, body composition, IGF-I levels and genetic differences (deletion / mUPD). |
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E.2.2 | Secondary objectives of the trial |
To assess oxytocin levels in blood and saliva samples before, during and after oxytocin treatment compared with placebo.
To evaluate the effect of intranasal oxytocin administration compared with placebo in relation to fMRI (BOLD response) in children >6 years. |
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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
- Age between 3 and 25 years
For fMRI: age > 6 years.
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E.4 | Principal exclusion criteria |
- Severe psychiatric problems
- Non cooperative behaviour
- Allergic reactions or hypersensitivity for oxytocin
- Serious illness
- Cardiac abnormalities
- 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) |
Change 4 weeks oxytocin versus 4 week placebo on:
- Body composition (Anthropometric measurements, BMI and DXA-scan)
- Eating behaviour (Dykens hyperphagia questionnaire and questionnaire about behaviour)
- Social behaviour (Questionnaire about behaviour for parents and Theory of Mind test)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 1: Baseline assessments in the hospital, first administration of oxytocin/placebo followed by observation period and instruction of administration.
Day 2-28: Administration of oxytocin/placebo every morning and evening at home.
Day 29: Hospital visit and assessments after use of oxytocin/placebo for 4 weeks. First administration of the other drug (oxytocin/placebo) followed by observation period.
Day 30-56: Administration of oxytocin/placebo every morning and evening at home.
Day 57: Hospital visit and assessments after use of the other drug (oxytocin/placebo) for 4 weeks. |
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E.5.2 | Secondary end point(s) |
- Laboratory parameter (oxytocin in saliva and blood)
- Safety parameters (laboratory parameters and medical assessments).
- fMRI (BOLD responses) in children >6 years |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Day 1: Baseline assessments in the hospital, first administration of oxytocin/placebo followed by observation period and instruction of administration.
Day 2-28: Administration of oxytocin/placebo every morning and evening at home.
Day 29: Hospital visit and assessments after use of oxytocin/placebo for 4 weeks. First administration of the other drug (oxytocin/placebo) followed by observation period.
Day 30-56: Administration of oxytocin/placebo every morning and evening at home.
Day 57: Hospital visit and assessments after use of the other drug (oxytocin/placebo) for 4 weeks. |
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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) | 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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LVLS (last visit of the last subject) |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |