Easypod Connect: A National, Multicentre, Observational Registry to Study Adherence and Long Term Outcomes of Therapy in Paediatric Subjects Using Easypod™ Electromechanical Device for Growth Hormone Treatment
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 191
- 试验地点
- 1
- 主要终点
- Mean percent of adherence by subject over a period of time
研究概览
简要总结
This is a National, Multicentre, Observational Registry to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospital in the UK and to assess the level of adherence of subject receiving SAIZEN® via Easypod™.
详细描述
Subjects will be enrolled in a multicenter longitudinal observational registry. Parents/subjects will provide their Informed Consent/assent to upload their data for populationbased analyses and optionally to adhere to a patient adherence support program designed to improve their adherence and, ultimately, clinical outcomes. Adherence data will be primarily derived from the Easypod™ device combined with physician data entry of outcome measures. Data will be collected retrospectively and prospectively. This will allow the establishment of adherence profiles and explore the hypothesis that patient adherence support programs improve adherence and subsequent clinical outcomes. Collected data will be also analyzed in a multinational pooled analysis of comparable national studies.
Primary Objective:
- To assess the level of adherence of subjects receiving SAIZEN via easypod™
Secondary Objectives:
- To describe the impact of adherence on clinical outcomes for subject receiving SAIZEN via easypod™
- To identify adherence subject profiling
- To assess the impact of adherence on Insulin-like Growth Factor 1 (IGF-1) (i.e. above, below or within normal ranges)
- To assess the endocrinological profile including Triiodothyronine (T3), Thyroxine (T4), Thyroid-stimulating hormone (TSH), Insulin-like Growth Factor 1 (IGF-1) and Insulin-like Growth Factor-Binding Protein 3(IGFBP-3) levels
- Temporal profile of IGF-1 and IGFBP-3 levels
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Administered growth hormone via the easypod electromechanical injection device according to Summary of Product Characteristics (SmPC)
- •Over the age of 2 years
- •Under 18 years of age, or over 18 without fusion of growth plates (to be confirmed by the Investigator, where relevant)
- •Appropriate Informed Consent/Assent provided
排除标准
- •Patients taking growth hormone in whom growth plates have fused (i.e. for taking growth hormone for it's metabolic effects)
- •Contra-indications to Saizen as defined in the SmPC or any other condition which precludes the use of SAIZEN in a given patient
- •Use of an investigational drug or participation in an interventional clinical trial
结局指标
主要结局
Mean percent of adherence by subject over a period of time
时间窗: At least 6 months and up to 5 years
次要结局
- Subject adherence profile based on age, gender, indication, self-injection or not, time on treatment(At least 6 months and up to 5 years)
- Correlation of adherence and growth outcome (height velocity (HV), height velocity-standard deviation score (HV-SDS), height, change in height (height SDS) after each year of SAIZEN® treatment with easypod™(At least 6 months and up to 5 years)
- Impact of patient adherence support programme on adherence and outcomes for subjects using easypod™(At least 6 months and up to 5 years)