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

Prader-Willi Syndrome Clinical Trial

— OTBB3-FU  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05032326
• Other study ID #: RC31/20/0421
• Status: Recruiting
• Phase: Phase 3
• Start date: September 7, 2021
• Est. completion date: April 1, 2025

Study information

• Verified date: April 2024
• Source: University Hospital, Toulouse
• Contact: Maithé TAUBER, MD
• Phone: 534 55 85 51
• Email: tauber.mt[@]chu-toulouse.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: April 1, 2025
• Est. primary completion date: April 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 36 Months

Eligibility

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;

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Follow-up study of the treated cohort
follow-up study of the patients in the treated cohort: that have been included in the otbb3 study

Other:
• Follow-up study of the untreated cohort
follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study

Locations

Country	Name	City	State
France	Hôpital Femme Mère Enfant	Bron
France	CHU Dijon Hôpital des Enfants	Dijon
France	CHU de Grenoble	Grenoble
France	Hôpital Jeanne de Flandre	Lille
France	Hôpital de la Timone Enfant	Marseille
France	CHU Nancy	Nancy
France	CHU Nantes	Nantes
France	Hôpital CHU-Lenval	Nice
France	Groupe Hospitalier Necker - Enfants Malades	Paris
France	CHU Rennes	Rennes
France	CHU Rouen	Rouen
France	Centre de réfrence Prader-Willi, Hospital of infants	Toulouse

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Toulouse

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Confirmation of the long term safety profile (1)	The number of patients with adverse events (AEs)	4 years
Primary	Confirmation of the long term safety profile (2)	The percentage of patients with adverse events (AEs)	4 years
Primary	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
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Assessment of endocrine disorders by IGF1	Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)	4 years
Secondary	Assessment of endocrine disorders by TSH	Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)	4 years