A Phase 3 interventional study of Follow-up study of the treated cohort and Follow-up study of the untreated cohort in Prader-Willi Syndrome, sponsored by University Hospital, Toulouse. Recruiting at 12 sites in France. Open to participants aged 12 Months to 36 Months. Per ClinicalTrials.gov, last updated 2024-04-30.
Sponsored by University Hospital, Toulouse · Phase 3, Interventional, and Other
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.
138 studies on the registry are indexed under Prader-Willi Syndrome; 24 are open to participants now.
This study's planned enrollment of 80 is above the median of 30 across 96 interventional studies indexed under Prader-Willi Syndrome.
Browse Prader-Willi Syndrome studies →University Hospital, Toulouse is the lead sponsor of 794 studies on the registry; 214 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Administrative problems:
babies treated with Oxytocin during the OTBB3 study
Drug: Follow-up study of the treated cohort
babies not included in the OTBB3 study and therefore never treated with Oxytocin
Other: Follow-up study of the untreated cohort
follow-up study of the patients in the treated cohort: that have been included in the otbb3 study
follow-up study of the patients in the untreated cohort: that have NOT been included in the otbb3 study
Confirmation of the long term safety profile (1)
The number of patients with adverse events (AEs)
Time frame: 4 years
Confirmation of the long term safety profile (2)
The percentage of patients with adverse events (AEs)
Time frame: 4 years
Confirmation of the long term safety profile (3)
Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
Time frame: 4 years
Confirmation of the long term safety profile (4)
Assessment in the treated cohort of: The occurrence of medications, surgery and rehabilitations by collecting type, age (years) at start and stop, dosing or frequency
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (1.1)
Assessment in the treated cohort of: weight (kilograms)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (1.2)
Assessment in the treated cohort of: height (meters)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (1.3)
Assessment in the treated cohort of: BMI (kg/m\^2)
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.1)
Assessment in the treated cohort of Child development: age at which sitting has been reached
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.2)
Assessment in the treated cohort of Child development: age at which crawling has been reached
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.3)
Assessment in the treated cohort of Child development: age at which walking has been reached
Time frame: 4 years
Complete the safety assessment by the description of the development of the child (2.4)
Assessment in the treated cohort of Child development: age at which running has been reached
Time frame: 4 years
Complete the safety assessment by the description of the severity of the disease
Severity of the disease for: Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT);
Time frame: 4 years
Complete the safety assessment by the description of the severity of the disease (2)
Severity of the disease for: Psychiatric disorders by using the Child Behaviour Checklist (CBCL);
Time frame: 4 years
Assessment of endocrine disorders by IGF1
Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
Time frame: 4 years
Assessment of endocrine disorders by TSH
Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
Time frame: 4 years
Plan to share: Undecided
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University Hospital, Toulouse