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

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-006161-11-FR

Enrollment

Registered

2021-04-21

Start date

2021-06-23

Completion date

Unknown

Last updated

2025-01-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Prader-willi syndrome

Interventions

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

Eligibility

Sex/Gender

All

Inclusion 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

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;

Design outcomes

Primary

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

Measure

Time frame

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

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Secondary

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

Measure

Time frame

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

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Countries

France

Contacts

Public ContactTAUBER

Centre de référence Prader-Willi

tauber.mt@chu-toulouse.fr+330534558551

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026