E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Prader-willi syndrome |
Syndrome de Prader-Willi |
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E.1.1.1 | Medical condition in easily understood language |
Prader-Willi Syndrome |
Syndrome de Prader-Willi |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
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 confirm the long term safety profile including the associated comorbidities in all children with PWS who have been treated in the OTBB3 study. |
confirmer le profil de sécurité d’emploi à long terme, notamment les principales comorbidités associées, chez tous les enfants présentant un SPW ayant été traités au cours de l’étude OTBB3. |
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E.2.2 | Secondary objectives of the trial |
• To complete the safety assessment by describing the development of children and severity of the disease in all children included in the OTBB3 study; • To compare the long-term safety and efficacy of early OT treatment in children included in OTBB3 with an untreated cohort of French children with PWS. This comparison will be performed on the subset of patients who have been treated in France in the OTBB3 study in a comparable context.
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Compléter l’évaluation de la sécurité d’emploi en décrivant le développement des enfants et la sévérité de la maladie chez tous les enfants inclus dans l’étude OTBB3 ; Comparer la sécurité d’emploi et l’efficacité à long terme d’un traitement précoce par l’ocytocine chez les enfants inclus dans l’étude OTBB3, avec une cohorte non traitée d’enfants français présentant un SPW. Cette comparaison sera réalisée sur le sous-ensemble de patients ayant été traités en France dans l’étude OTBB3 dans un contexte comparable.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Male or female child with a genetically confirmed diagnosis of PWS The parents (or legal representative) must have signed the consent form; Treated cohort: the child participated in the OTBB3 study Untreated cohort: the child has never received OT, is aged 30±6 months at inclusion and is followed in France.
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1. Enfant de sexe masculin ou féminin présentant un diagnostic génétiquement confirmé de SPW 2. Les parents (ou le représentant légal) doivent avoir signé le formulaire de consentement ; 3. Cohorte traitée : l’enfant a participé à l’étude OTBB3 ; 4. Cohorte non traitée : l’enfant n’a jamais reçu d’ocytocine, est âgé de 30±6 mois à l’inclusion et est suivi en France.
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E.4 | Principal exclusion criteria |
1. Administrative problems: a. Inability for the parents (or legal representative) to understand/fulfil study requirements; b. No coverage by a social security regime; 2. Refusal of parents (or legal representative) to sign the consent form;
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1. Problèmes administratifs : a. Incapacité des parents (ou du représentant légal) à comprendre/respecter les exigences de l’étude ; b. Absence de couverture par un régime de sécurité sociale ; 2. Refus des parents (ou du représentant légal) de signer le formulaire de consentement ;
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E.5 End points |
E.5.1 | Primary end point(s) |
• The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs); • The occurrence of the following main comorbidities: o Digestive disorders o Scoliosis; o Sleep disorders; - Obstructive and central sleep apnoea - Narcolepsy; - Excessive daytime sleepiness o Endocrine disorders o Metabolic disorders o Other comorbidities assessed by a checklist. • The occurrence of medications, surgery and rehabilitations |
• Nombre et pourcentage de patients présentant des événements indésirables (EI) et des événements indésirables graves (EIG) ; • Survenue des principales comorbidités suivantes : o Troubles digestifs o Scoliose ; o Troubles du sommeil ; - Apnée obstructive et apnée centrale du sommeil - Narcolepsie ; - Somnolence diurne excessive o Troubles endocriniens o Troubles métaboliques o Autres comorbidités évalués par une liste de contrôle. • Prise de médicaments, chirurgie et rééducation
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)
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chaque année en V1, V2 et V3 entre 36 et 48 mois (42 +/- 6 mois) |
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E.5.2 | Secondary end point(s) |
• The number and percentage of patients with AEs and SAEs; • The occurrence of the following main comorbidities: o Digestive disorders o Scoliosis; o Sleep disorders o Endocrine disorders o Metabolic disorders o Other comorbidities • The occurrence of medications, surgery and rehabilitations • The severity of the comorbidities • Auxology, anthropometry and psychomotor development • Severity of the disease in terms of: o Miller nutritional phases; o Eating behaviour o Adaptive behaviour o Behavioural and psychiatric disorders • Caregiver burden
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• Nombre et pourcentage de patients présentant des EI et des EIG ; • Survenue des principales comorbidités suivantes : o Troubles digestifs o Scoliose ; o Troubles du sommeil o Troubles endocriniens o Troubles métaboliques o Autres comorbidités • Prise de médicaments, chirurgie et rééducation • Sévérité des comorbidités • Auxologie, anthropométrie et développement psychomoteur • Sévérité de la maladie en utilisant les critères suivants : o Phases nutritionnelles de Miller ; o Comportement alimentaire o Comportement adaptatif o Troubles comportementaux et psychiatriques • Charge pesant sur les aidants |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months) |
chaque année en V1, V2 et V3 entre 36 et 48 mois (42 +/- 6 mois) |
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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 | No |
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 | No |
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 | Yes |
E.8.1.6 | Cross over | No |
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 | Yes |
E.8.2.3.1 | Comparator description |
children untreated by oxytocin |
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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
| 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 | 14 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
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 |
Dernière visite du dernier patient |
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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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |