Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial

Study Overview
=================
Brief Summary
-----------------
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.

Official Title
-----------------
Long-term Interventional Follow-up Study up to 4 Years of Age of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial and Comparison With an Untreated Cohort of Children With Prader-Willi Syndrome

Conditions
-----------------
Prader-Willi Syndrome

Intervention / Treatment
-----------------
* Drug: Follow-up study of the treated cohort
* Other: Follow-up study of the untreated cohort


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Male or female child with a genetically confirmed diagnosis of PWS (patients can be enrolled if the genetic subtype is not available at inclusion, but the genetic subtype needs to be confirmed during the study); The parents (or legal representative) must have signed the consent form; Treated cohort: the child participated in the OTBB3 study and is aged 16±4 months at inclusion, Untreated cohort: the child has never received OT, is aged 30±6 months at inclusion (in order to maximise the number of children in the untreated cohort) and is followed in France. Exclusion Criteria: Administrative problems: Inability for the parents (or legal representative) to understand/fulfil study requirements; No coverage by a social security regime; Refusal of parents (or legal representative) to sign the consent form;

Ages Eligible for Study
-----------------
Minimum Age: 12 Months
Maximum Age: 36 Months

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Other
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: OXYTOCIN (OT) Treated cohort<br>babies treated with Oxytocin during the OTBB3 study | Drug: Follow-up study of the treated cohort<br>* follow-up study of the patients in the treated cohort: that have been included in the otbb3 study<br>|
| Other: Untreated cohort<br>babies not included in the OTBB3 study and therefore never treated with Oxytocin | Other: Follow-up study of the untreated cohort<br>* follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | 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 | 

Secondary Outcome Measures

| Outcome Measure | 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) | 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Prader-Willi syndrome, Oxytocin