Long-term follow-up of children with Prader-Willi Syndrome

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

This study is tracking children with Prader-Willi Syndrome over four years to see how their condition changes, comparing those who received oxytocin treatment to those who did not.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 80 (estimated)
• Ages: 12 Months to 36 Months
• Sex: All
• Sponsor: University Hospital, Toulouse (Academic / other)
• Locations: 12 sites (Bron and 11 other locations)
• Trial ID: NCT05032326 on ClinicalTrials.gov

What this trial studies

This study is a prospective, multicenter, interventional cohort study focusing on children with Prader-Willi Syndrome (PWS) over a period of four years. It includes both a treated cohort, who participated in the previous OTBB3 trial, and an untreated cohort of children who have not received oxytocin treatment. The study aims to follow these children from the age of 2 to 4 years to gather long-term data on their condition. The untreated cohort will consist of children born in France who were too old to be included in the OTBB3 trial.

Who should consider this trial

Good fit: Ideal candidates are children aged 30±6 months with a genetically confirmed diagnosis of Prader-Willi Syndrome who have never received oxytocin treatment.

Not a fit: Patients who have previously received oxytocin treatment or do not meet the age and genetic criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term effects of oxytocin treatment in children with Prader-Willi Syndrome.

How similar studies have performed: Other studies have shown promise in the treatment of Prader-Willi Syndrome with oxytocin, making this approach potentially beneficial.

Eligibility criteria

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;

Where this trial is running

Bron and 11 other locations

• 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, France (Recruiting)

Study contacts

• Principal investigator: Maithé TAUBER, MD — University Hospital, Toulouse
• Study coordinator: Maithé TAUBER, MD

Conditions Prader-Willi Syndrome; Prader-Willi syndrome; Oxytocin