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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 80
- 试验地点
- 12
- 主要终点
- Confirmation of the long term safety profile (2)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 36 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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;
研究组 & 干预措施
OXYTOCIN (OT) Treated cohort
babies treated with Oxytocin during the OTBB3 study
干预措施: Follow-up study of the treated cohort (Drug)
Untreated cohort
babies not included in the OTBB3 study and therefore never treated with Oxytocin
干预措施: Follow-up study of the untreated cohort (Other)
结局指标
主要结局
Confirmation of the long term safety profile (2)
时间窗: 4 years
The percentage of patients with adverse events (AEs)
Confirmation of the long term safety profile (3)
时间窗: 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)
时间窗: 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
Confirmation of the long term safety profile (1)
时间窗: 4 years
The number of patients with adverse events (AEs)
次要结局
- Complete the safety assessment by the description of the development of the child (1.2)(4 years)
- Complete the safety assessment by the description of the development of the child (1.3)(4 years)
- Complete the safety assessment by the description of the development of the child (1.1)(4 years)
- Complete the safety assessment by the description of the development of the child (2.3)(4 years)
- Assessment of endocrine disorders by IGF1(4 years)
- Assessment of endocrine disorders by TSH(4 years)
- Complete the safety assessment by the description of the development of the child (2.2)(4 years)
- Complete the safety assessment by the description of the development of the child (2.4)(4 years)
- Complete the safety assessment by the description of the severity of the disease (2)(4 years)
- Complete the safety assessment by the description of the development of the child (2.1)(4 years)
- Complete the safety assessment by the description of the severity of the disease(4 years)