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

Toulouse University Hospital

Status and phase: Enrolling; Phase 3

Conditions: Prader-Willi Syndrome

Treatments: Drug: Follow-up study of the treated cohort; Other: Follow-up study of the untreated cohort

Study type: Interventional

Funder types: Other

Identifiers

NCT05032326

RC31/20/0421

Details and patient eligibility

About

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.

Enrollment: 80 estimated patients

Sex: All

Ages: 12 to 36 months old

Volunteers: No Healthy Volunteers

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;

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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80 participants in 2 patient groups

OXYTOCIN (OT) Treated cohort

Other group

Description:

babies treated with Oxytocin during the OTBB3 study

Treatment:

Drug: Follow-up study of the treated cohort

Untreated cohort

Other group

Description:

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

Treatment:

Other: Follow-up study of the untreated cohort