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

University Hospital, Toulouse80 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

OTHER

Masking

NONE

Enrollment

Conditions

Prader-Willi Syndrome

Interventions

Eligibility

Sex: ALLMin age: 12 MonthsMax age: 36 Months

Medical Language ↔ Plain English

Inclusion Criteria: 1. 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); 2. The parents (or legal representative) must have signed the consent form; 3. Treated cohort: the child participated in the OTBB3 study and is aged 16±4 months at inclusion, 4. 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: 1. Administrative problems: 1. Inability for the parents (or legal representative) to understand/fulfil study requirements; 2. No coverage by a social security regime; 2. Refusal of parents (or legal representative) to sign the consent form;

Locations (12)

Hôpital Femme Mère Enfant

Bron, France

RECRUITING

CHU Dijon Hôpital des Enfants

Dijon, France

RECRUITING

CHU de Grenoble

Grenoble, France

RECRUITING

Hôpital Jeanne de Flandre

Lille, France

RECRUITING

Hôpital de la Timone Enfant

Marseille, France

RECRUITING

CHU Nancy

Nancy, France

RECRUITING

CHU Nantes

Nantes, France

RECRUITING

Hôpital CHU-Lenval

Nice, France

RECRUITING

Groupe Hospitalier Necker - Enfants Malades

Paris, France

RECRUITING

CHU Rennes

Rennes, France

RECRUITING

...and 2 more locations

Outcomes

Primary Outcomes

Confirmation of the long term safety profile (1)

The number of patients with adverse events (AEs)

Time frame: 4 years

Confirmation of the long term safety profile (2)

The percentage of patients with adverse events (AEs)

Time frame: 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

Time frame: 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

Time frame: 4 years

Secondary Outcomes

Complete the safety assessment by the description of the development of the child (1.1)

Assessment in the treated cohort of: weight (kilograms)

Time frame: 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)

Time frame: 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)

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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);

Time frame: 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);

Time frame: 4 years

Assessment of endocrine disorders by IGF1

Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)

Time frame: 4 years

Assessment of endocrine disorders by TSH

Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)

Time frame: 4 years

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

Overview

Conditions

Interventions

Eligibility

Locations (12)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts