FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL

• Register: EUCTR
• Last refreshed on: 7 January 2025
• Main ID: EUCTR2020-006161-11-FR
• Date of registration: 21/04/2021
• Prospective Registration: Yes
• Primary sponsor: Toulouse University Hospital
• Public title: FOLLOW-UP STUDY UP TO 4 YEARS OF AGE OF CHILDREN WITH PRADER-WILLI SYNDROME INCLUDED IN THE OTBB3 CLINICAL TRIAL
• Scientific 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 - OTBB3 Follow-Up
• Date of first enrolment: 23/06/2021
• Target sample size: 80
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-006161-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: children untreated by oxytocin Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

France

Contacts

• Name: TAUBER
• Affiliation: Centre de référence Prader-Willi

• Name: TAUBER
• Affiliation: Centre de référence Prader-Willi

Key inclusion & exclusion criteria

Inclusion criteria:
Male or female child with a genetically confirmed diagnosis of PWS
The parents (or legal representative) must have signed the consent form;
Treated cohort: the child participated in the OTBB3 study
Untreated cohort: the child has never received OT, is aged 30±6 months at inclusion and is followed in France.

Are the trial subjects under 18? yes
Number of subjects for this age range: 80
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Administrative problems:
a. Inability for the parents (or legal representative) to understand/fulfil study requirements;
b. No coverage by a social security regime;
2. Refusal of parents (or legal representative) to sign the consent form;




Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Prader-willi syndrome

Intervention(s)

Product Name: oxytocin
Pharmaceutical Form: Nasal spray, solution
Pharmaceutical form of the placebo: Nasal spray
Route of administration of the placebo: Nasal use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)

Primary end point(s): •The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
•The occurrence of the following main comorbidities:
oDigestive disorders
oScoliosis;
oSleep disorders;
-Obstructive and central sleep apnoea
-Narcolepsy;
-Excessive daytime sleepiness
oEndocrine disorders
oMetabolic disorders
oOther comorbidities assessed by a checklist.
•The occurrence of medications, surgery and rehabilitations

Secondary Objective: •To complete the safety assessment by describing the development of children and severity of the disease in all children included in the OTBB3 study;
•To compare the long-term safety and efficacy of early OT treatment in children included in OTBB3 with an untreated cohort of French children with PWS. This comparison will be performed on the subset of patients who have been treated in France in the OTBB3 study in a comparable context.

Main Objective: To confirm the long term safety profile including the associated comorbidities in all children with PWS who have been treated in the OTBB3 study.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: every year at V1, V2 and V3 between 36 to 48 months (42+/-6 months)

Secondary end point(s): • The number and percentage of patients with AEs and SAEs;
• The occurrence of the following main comorbidities:
o Digestive disorders
o Scoliosis;
o Sleep disorders
o Endocrine disorders
o Metabolic disorders
o Other comorbidities
• The occurrence of medications, surgery and rehabilitations
• The severity of the comorbidities
• Auxology, anthropometry and psychomotor development
• Severity of the disease in terms of:
o Miller nutritional phases;
o Eating behaviour
o Adaptive behaviour
o Behavioural and psychiatric disorders
• Caregiver burden

Secondary ID(s)

RC31/20/0421

Source(s) of Monetary Support

OT4B

Ethics review

Status: Approved
Approval date: 09/06/2021
Contact: