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 | 19.0 |
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 long-term intranasal oxytocin on social behaviour in children with PWS - To evaluate the effects of long-term intranasal oxytocin administration on appetite, satiety, food intake and food seeking behaviour in children with PWS - To investigate if there is a difference in the efficacy of oxytocin if adminstered in different doses and frequencies |
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E.2.2 | Secondary objectives of the trial |
To evaluate oxytocin levels in blood and saliva samples before, during and after intranasal oxytocin treatment in different doses and frequencies in children with PWS - To evaluate the effects of long-term intranasal oxytocin administration in different doses and frequencies in relation to: BMI, IGF-1 and IGF-BP3 levels, fMRI (BOLD response)
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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 16 years - Currently on growth hormone treatment for at least 1 year - Behavioural problems (for example temper tantrums and autistic behaviour) and/or be in nutritional phase 2b or 3 according to Miller.
For fMRI: age > 6 years |
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E.4 | Principal exclusion criteria |
- Severe psychiatric problems - Non-cooperatieve behaviour - Allergic reactions or hypersensitivity for oxytocin - Serious illness - Cardiac abnormalities - Extremely low dietary intake or less than minimal required intake 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 and food related behaviour assessed by: - Oxytocin Study Questionnaire - VISK |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Studygroup 1: Day 1: baseline assessments in the hospital, first administration of oxytocin followed by observation period and instruction of administration Day 2-month 6: administration of oxytocin one/twice a day every day at home - 6 months: hospital visit and assessments as mentioned on day 1 - Mont 6-month 7: wash-out period - 7 months: parents fill out a questionnaire at home. Start other dosing regimen. Month 7-month 13: administration of oxytocin once/twice daily every day at home 13 months: hospital visit and assessment as mentioned on day 1
For studygroup 2 assessments will be done on day 1 (hospital), at 3 months (hospital), at 4 months (questionnaire at home), 7 months (hospital)
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E.5.2 | Secondary end point(s) |
Change in: - Body composition - Quality of life - Hyperphagia questionnaire - Theory of Mind test - Diary concerning social and food related behaviour - fMRI (>6 years old) - Laboratory parameters - Safety parameters |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Studygroup 1: Day 1: baseline assessments in the hospital, first administration of oxytocin followed by observation period and instruction of administration Day 2-month 6: administration of oxytocin one/twice a day every day at home - 6 months: hospital visit and assessments as mentioned on day 1 - Mont 6-month 7: wash-out period - 7 months: parents fill out a questionnaire at home. Start other dosing regimen. Month 7-month 13: administration of oxytocin once/twice daily every day at home 13 months: hospital visit and assessment as mentioned on day 1
For studygroup 2 assessments will be done on day 1 (hospital), at 3 months (hospital), at 4 months (questionnaire at home), 7 months (hospital)
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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) | 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 | Yes |
E.8.1.2 | Open | Yes |
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 | 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 | No |
E.8.2.3 | Other | No |
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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |