Clinical trial NCT05032326: Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Keywords: Prader-Willi syndrome
Oxytocin
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
First posted: 2021-09-02
Enrollment: 80
Sex: ALL
Minimum age (years): 12
Maximum age (years): 36
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.


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;

Primary outcomes:
measure: Confirmation of the long term safety profile (1)
timeFrame: 4 years
measure: Confirmation of the long term safety profile (2)
timeFrame: 4 years
measure: Confirmation of the long term safety profile (3)
timeFrame: 4 years
measure: Confirmation of the long term safety profile (4)
timeFrame: 4 years
Secondary outcomes:
measure: Complete the safety assessment by the description of the development of the child (1.1)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the development of the child (1.2)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the development of the child (1.3)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the development of the child (2.1)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the development of the child (2.2)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the development of the child (2.3)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the development of the child (2.4)
timeFrame: 4 years
measure: Complete the safety assessment by the description of the severity of the disease
timeFrame: 4 years
measure: Complete the safety assessment by the description of the severity of the disease (2)
timeFrame: 4 years
measure: Assessment of endocrine disorders by IGF1
timeFrame: 4 years
measure: Assessment of endocrine disorders by TSH
timeFrame: 4 years
Other outcomes:
Locations:
city: Bron
country: France
facility: Hôpital Femme Mère Enfant
city: Dijon
country: France
facility: CHU Dijon Hôpital des Enfants
city: Grenoble
country: France
facility: CHU de Grenoble
city: Lille
country: France
facility: Hôpital Jeanne de Flandre
city: Marseille
country: France
facility: Hôpital de la Timone Enfant
city: Nancy
country: France
facility: CHU Nancy
city: Nantes
country: France
facility: CHU Nantes
city: Nice
country: France
facility: Hôpital CHU-Lenval
city: Paris
country: France
facility: Groupe Hospitalier Necker - Enfants Malades
city: Rennes
country: France
facility: CHU Rennes
city: Rouen
country: France
facility: CHU Rouen
city: Toulouse
country: France
facility: Centre de réfrence Prader-Willi, Hospital of infants
Study officials:
affiliation: University Hospital, Toulouse
name: Maithé TAUBER, MD
role: PRINCIPAL_INVESTIGATOR
References: