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

Phase 3

Recruiting

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Follow-up study of the treated cohort; Other: Follow-up study of the untreated cohort

• Registration Number: NCT05032326
• Lead Sponsor: University Hospital, Toulouse

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-06-24
• Study First Submitted QC: 2021-08-26
• Study First Posted: 2021-09-02
• Study Start: 2021-09-07
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-29
• Last Update Posted: 2024-04-30
• Primary Completion: 2025-04-01
• Study Completion: 2025-04-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 80

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;

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
OXYTOCIN (OT) Treated cohort	Follow-up study of the treated cohort	babies treated with Oxytocin during the OTBB3 study
Untreated cohort	Follow-up study of the untreated cohort	babies not included in the OTBB3 study and therefore never treated with Oxytocin

Primary Outcome Measures

Name	Time	Method
Confirmation of the long term safety profile (1)	4 years	The number of patients with adverse events (AEs)
Confirmation of the long term safety profile (2)	4 years	The percentage of patients with adverse events (AEs)
Confirmation of the long term safety profile (3)	4 years	Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
Confirmation of the long term safety profile (4)	4 years	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

Secondary Outcome Measures

Name	Time	Method
Complete the safety assessment by the description of the development of the child (1.2)	4 years	Assessment in the treated cohort of: height (meters)
Complete the safety assessment by the description of the development of the child (1.1)	4 years	Assessment in the treated cohort of: weight (kilograms)
Complete the safety assessment by the description of the development of the child (1.3)	4 years	Assessment in the treated cohort of: BMI (kg/m\^2)
Complete the safety assessment by the description of the development of the child (2.3)	4 years	Assessment in the treated cohort of Child development: age at which walking has been reached
Assessment of endocrine disorders by IGF1	4 years	Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
Assessment of endocrine disorders by TSH	4 years	Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
Complete the safety assessment by the description of the development of the child (2.2)	4 years	Assessment in the treated cohort of Child development: age at which crawling has been reached
Complete the safety assessment by the description of the development of the child (2.4)	4 years	Assessment in the treated cohort of Child development: age at which running has been reached
Complete the safety assessment by the description of the severity of the disease (2)	4 years	Severity of the disease for: Psychiatric disorders by using the Child Behaviour Checklist (CBCL);
Complete the safety assessment by the description of the development of the child (2.1)	4 years	Assessment in the treated cohort of Child development: age at which sitting has been reached
Complete the safety assessment by the description of the severity of the disease	4 years	Severity of the disease for: Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT);

Trial Locations

Locations (12)

Hôpital Femme Mère Enfant; 🇫🇷 Bron, France

CHU Dijon Hôpital des Enfants; 🇫🇷 Dijon, France

CHU de Grenoble; 🇫🇷 Grenoble, France

Hôpital de la Timone Enfant; 🇫🇷 Marseille, France

CHU Rennes; 🇫🇷 Rennes, France

CHU Nantes; 🇫🇷 Nantes, France

Hôpital Jeanne de Flandre; 🇫🇷 Lille, France

CHU Nancy; 🇫🇷 Nancy, France

Hôpital CHU-Lenval; 🇫🇷 Nice, France

Groupe Hospitalier Necker - Enfants Malades; 🇫🇷 Paris, France

CHU Rouen; 🇫🇷 Rouen, France

Centre de réfrence Prader-Willi, Hospital of infants; 🇫🇷 Toulouse, France