E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Prader-Willi syndrom |
Syndrome de Prader-Willi |
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E.1.1.1 | Medical condition in easily understood language |
Prader-Willi syndrom |
syndrome de Prader-Willi |
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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 | 18.0 |
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 |
Etudier l’efficacité de l’administration intranasale répétée d’OT pendant un mois (selon 2 modalités d’administration, quotidienne ou bihebdomadaire) en comparaison à un placebo |
Determinate efficacy of nasal administration of ocytocine during one month by comparing with placebo. |
|
E.2.2 | Secondary objectives of the trial |
Etudier l’efficacité de l’administration intranasale répétée d’OT pendant un mois (selon 2 modalités d’administration, quotidienne ou bihebdomadaire) en comparaison à un placebo, chez des adultes présentant un SPW, sur les compétences sociales, la pragmatique, les fonctions exécutives, les compétences neurovisuelles et la reconnaissance vocale, le métabolisme cérébral et sur les taux circulants d’OT et de ghreline. Comparer l’efficacité des 2 schémas d’administration. |
Determinate efficacy of nasal administration of ocytocine during one month by comparing with placebo on social relations, executives actions, visions, vocal recognizing. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients adultes de sexe féminin ou masculin présentant un syndrome de Prader-Willi :
- Diagnostic confirmé génétiquement et permettant de savoir s’il s’agit d’une délétion ou non
-Age : à partir de 18 ans et inférieur à 40 ans |
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E.4 | Principal exclusion criteria |
Patients présentant des troubles psychiatriques non équilibrés
Patients présentant des anomalies du rythme cardiaque à l’ECG significatifs après avis d’un cardiologue
- Patients présentant une insuffisance hépatique
- Patients présentant une insuffisance rénale
- Patientes présentant une grossesse ou allaitante
- Patients présentant une hypersensibilité à l’OT
- Patients traités par des médicaments (pendant la durée de l’étude) susceptibles d’entraîner des troubles du rythme (notamment les B-bloquants)
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E.5 End points |
E.5.1 | Primary end point(s) |
grilles d'évaluation du comportement |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
J1, J2à J3, J6 à J32, J34. |
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E.5.2 | Secondary end point(s) |
échelles analogiques, IRM, TEP |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
J1, J2à J3, J6 à J32, J34.
TEP et IRM à J4, J5 et J35.
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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 | No |
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 | 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 | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
2 brais traitement avec doses différentes + un bras placebo |
|
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 | 3 |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |