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)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 65
- 试验地点
- 11
- 主要终点
- The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
研究概览
简要总结
The primary objective of this study will be to confirm the long-term safety profile, including the associated main comorbidities, in all children with PWS who have been treated in the OTBB3 study.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •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 and is aged 16±4 months at inclusion. Inclusion visit will be performed considering the next routine consultation.
- •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. Inclusion visit will be performed considering the next routine consultation.
- •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. Inclusion visit will be performed considering the next routine consultation.
排除标准
- •Administrative problems: a. Inability for the parents (or legal representative) to understand/fulfil study requirements; b. No coverage by a social security regime;
- •Refusal of parents (or legal representative) to sign the consent form;
结局指标
主要结局
The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
The number and percentage of patients with adverse events (AEs) and serious adverse events (SAEs);
The occurrence of the main comorbidities
The occurrence of the main comorbidities
The occurrence of medications, surgery and rehabilitations by collecting type, age at start and stop, dosing or frequency.
The occurrence of medications, surgery and rehabilitations by collecting type, age at start and stop, dosing or frequency.
次要结局
未报告次要终点
研究者
TAUBER
Scientific
Centre Hospitalier Universitaire De Toulouse