OTBB3-FU: Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial
Study Details
Study Description
Brief Summary
This study is a prospective, multicentre, interventional cohort study in children with 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Other: OXYTOCIN Treated cohort babies treated with Oxytocin during the OTBB3 study |
Drug: Follow-up study of the treated cohort
follow-up study of the patients in the treated cohort: that have been included in the otbb3 study
|
| Other: Untreated 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 untreated cohort: that have NOT been included in the otbb3 study
|
Outcome Measures
Primary Outcome Measures
- Confirmation of the long term safety profile (1) [4 years]
The number of patients with adverse events (AEs)
- Confirmation of the long term safety profile (2) [4 years]
The percentage of patients with adverse events (AEs)
- Confirmation of the long term safety profile (3) [4 years]
Assessment in the treated cohort of the occurrence of the main comorbidities in Prader Willi Syndrome
- Confirmation of the long term safety profile (4) [4 years]
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
Secondary Outcome Measures
- Complete the safety assessment by the description of the development of the child (1.1) [4 years]
Assessment in the treated cohort of: weight (kilograms)
- Complete the safety assessment by the description of the development of the child (1.2) [4 years]
Assessment in the treated cohort of: height (meters)
- Complete the safety assessment by the description of the development of the child (1.3) [4 years]
Assessment in the treated cohort of: BMI (kg/m^2)
- Complete the safety assessment by the description of the development of the child (2.1) [4 years]
Assessment in the treated cohort of Child development: age at which sitting has been reached
- Complete the safety assessment by the description of the development of the child (2.2) [4 years]
Assessment in the treated cohort of Child development: age at which crawling has been reached
- Complete the safety assessment by the description of the development of the child (2.3) [4 years]
Assessment in the treated cohort of Child development: age at which walking has been reached
- Complete the safety assessment by the description of the development of the child (2.4) [4 years]
Assessment in the treated cohort of Child development: age at which running has been reached
- Complete the safety assessment by the description of the severity of the disease [4 years]
Severity of the disease for: Eating disorders by using Hyperphagia Questionnaire for Clinical Trials (HQCT);
- Complete the safety assessment by the description of the severity of the disease (2) [4 years]
Severity of the disease for: Psychiatric disorders by using the Child Behaviour Checklist (CBCL);
- Assessment of endocrine disorders by IGF1 [4 years]
Analysis of plasma Insulin-like growth factor 1 (IGF1, ng/mL)
- Assessment of endocrine disorders by TSH [4 years]
Analysis of plasma thyroid stimulating hormone (TSH, µUI/mL)
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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);
-
The parents (or legal representative) must have signed the consent form;
-
Treated cohort: the child participated in the OTBB3 study and is aged 16±4 months at inclusion,
-
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:
-
Administrative problems:
-
Inability for the parents (or legal representative) to understand/fulfil study requirements;
-
No coverage by a social security regime;
-
Refusal of parents (or legal representative) to sign the consent form;
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Centre de réfrence Prader-Willi, Hospital of infants | Toulouse | France | 31059 |
Sponsors and Collaborators
- University Hospital, Toulouse
Investigators
- Principal Investigator: Maithé TAUBER, MD, University Hospital, Toulouse
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- RC31/20/0421