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

NCT05032326 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: RC31/20/0421
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 12

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.

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi syndrome; Oxytocin

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (11)

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

  4 years

• Complete the safety assessment by the description of the development of the child (1.2)

  4 years

• Complete the safety assessment by the description of the development of the child (1.3)

  4 years

• Complete the safety assessment by the description of the development of the child (2.1)

  4 years

• Complete the safety assessment by the description of the development of the child (2.2)

  4 years

Study Arms (2)

OXYTOCIN (OT) Treated cohort

OTHER

babies treated with Oxytocin during the OTBB3 study

Drug: Follow-up study of the treated cohort

Untreated cohort

OTHER

babies not included in the OTBB3 study and therefore never treated with Oxytocin

Other: Follow-up study of the untreated cohort

Interventions

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

OXYTOCIN (OT) Treated cohort

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

Untreated cohort

Eligibility Criteria

• Age: 12 Months - 36 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• The parents (or legal representative) must have signed the consent form;

You may not qualify if:

• 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;

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (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

CHU Rouen

Rouen, France

RECRUITING

Centre de réfrence Prader-Willi, Hospital of infants

Toulouse, 31059, France

RECRUITING

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Officials

• Maithé TAUBER, MD

  University Hospital, Toulouse

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Maithé TAUBER, MD

CONTACT

534 55 85 51; tauber.mt@chu-toulouse.fr

Julie CORTADELLAS

CONTACT

534 55 85 51; cortadellas.j@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 24, 2021
• First Posted: September 2, 2021
• Study Start: September 7, 2021
• Primary Completion: April 1, 2025
• Study Completion: April 1, 2025
• Last Updated: April 30, 2024

Record last verified: 2024-04

Locations

FR (12)