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This study is a prospective, multicentre, interventional cohort study in children with Prader-Willi Syndrome (PWS) over 4 years (no treatment administered). The duration of the preceding OTTB3 study is 26 weeks. An untreated cohort of children with PWS will be included at an age of 2 years and followed up until an age of 4 years.
Regarding the untreated cohort, children with PWS born in France and too old to be recruited in OTBB3 trial, principally those who were born within one year before the start of OTBB3 trial, will be offered to participate in this study. Infants born later who couldn't be included in OTBB3 study will be also offered to participate.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OXYTOCIN (OT) Treated cohort | Other | babies treated with Oxytocin during the OTBB3 study |
|
| Untreated cohort | Other | babies not included in the OTBB3 study and therefore never treated with Oxytocin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Follow-up study of the treated cohort | Drug | follow-up study of the patients in the treated cohort: that have been included in the otbb3 study |
|
| Measure | Description | Time Frame |
|---|---|---|
| Confirmation of the long term safety profile (1) | The number of patients with adverse events (AEs) | 4 years |
| Confirmation of the long term safety profile (2) | The percentage of patients with adverse events (AEs) | 4 years |
| Confirmation of the long term safety profile (3) | Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome | 4 years |
| Confirmation of the long term safety profile (4) | Assessment in the treated cohort of: The occurrence of medications, surgery and rehabilitations by collecting type, age (years) at start and stop, dosing or frequency | 4 years |
| Measure | Description | Time Frame |
|---|---|---|
| Complete the safety assessment by the description of the development of the child (1.1) | Assessment in the treated cohort of: weight (kilograms) | 4 years |
| Complete the safety assessment by the description of the development of the child (1.2) |
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Inclusion Criteria:
Exclusion Criteria:
Administrative problems:
Refusal of parents (or legal representative) to sign the consent form;
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Maithé TAUBER, MD | Contact | 534 55 85 51 | +33 | tauber.mt@chu-toulouse.fr |
| Julie CORTADELLAS | Contact | 534 55 85 51 | +33 | cortadellas.j@chu-toulouse.fr |
| Name | Affiliation | Role |
|---|---|---|
| Maithé TAUBER, MD | University Hospital, Toulouse | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Femme Mère Enfant | Recruiting | Bron | France |
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| Follow-up study of the untreated cohort | Other | follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study |
|
Assessment in the treated cohort of: height (meters)
| 4 years |
| Complete the safety assessment by the description of the development of the child (1.3) | Assessment in the treated cohort of: BMI (kg/m^2) | 4 years |
| Complete the safety assessment by the description of the development of the child (2.1) | Assessment in the treated cohort of Child development: age at which sitting has been reached | 4 years |
| Complete the safety assessment by the description of the development of the child (2.2) | Assessment in the treated cohort of Child development: age at which crawling has been reached | 4 years |
| Complete the safety assessment by the description of the development of the child (2.3) | Assessment in the treated cohort of Child development: age at which walking has been reached | 4 years |
| Complete the safety assessment by the description of the development of the child (2.4) | Assessment in the treated cohort of Child development: age at which running has been reached | 4 years |
| Complete the safety assessment by the description of the severity of the disease | Severity of the disease for: Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT); | 4 years |
| Complete the safety assessment by the description of the severity of the disease (2) | Severity of the disease for: Psychiatric disorders by using the Child Behaviour Checklist (CBCL); | 4 years |
| Assessment of endocrine disorders by IGF1 | Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL) | 4 years |
| Assessment of endocrine disorders by TSH | Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL) | 4 years |
| CHU Dijon Hôpital des Enfants | Recruiting | Dijon | France |
|
| CHU de Grenoble | Recruiting | Grenoble | France |
|
| Hôpital Jeanne de Flandre | Recruiting | Lille | France |
|
| Hôpital de la Timone Enfant | Recruiting | Marseille | France |
|
| CHU Nancy | Recruiting | Nancy | France |
|
| CHU Nantes | Recruiting | Nantes | France |
|
| Hôpital CHU-Lenval | Recruiting | Nice | France |
|
| Groupe Hospitalier Necker - Enfants Malades | Recruiting | Paris | France |
|
| CHU Rennes | Recruiting | Rennes | France |
|
| CHU Rouen | Recruiting | Rouen | France |
|
| Centre de réfrence Prader-Willi, Hospital of infants | Recruiting | Toulouse | 31059 | France |
|
| ID | Term |
|---|---|
| D011218 | Prader-Willi Syndrome |
| ID | Term |
|---|---|
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
| D009765 | Obesity |
| D050177 | Overweight |
| D044343 | Overnutrition |
| D009748 | Nutrition Disorders |
| D009750 | Nutritional and Metabolic Diseases |
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