Open-label, Long-term Follow-up of Safety and Biochemical Disease Control of Infacort® in Neonates, Infants and Children With Congenital Adrenal Hyperplasia and Adrenal Insufficiency Previously Enrolled in the Infacort 003 Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)
研究概览
简要总结
A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with adrenal insufficiency and congenital adrenal hyperplasia who had completed study Infacort 003. All subjects who had satisfactorily completed study Infacort 003 were offered the opportunity to take part in Infacort 004.
详细描述
A Phase 3, open-label, single-group, non-randomised, observational study of the safety and biochemical disease control of Infacort® in neonates, infants and children with AI who had completed study Infacort 003 (EudraCT number 2014-002265-30). All subjects who had satisfactorily completed study Infacort 003 wiere offered the opportunity to participate in study Infacort 004 at or after their final visit of study Infacort 003. Subjects received the usual clinically-appropriate dose (since bioequivalence has been demonstrated with conventional hydrocortisone), as determined by the Investigator, which was administered according to usual clinical practice - generally 3 or 4 times a day. Subjects could continue to be treated in this study until they met the study withdrawal criteria, until Infacort® was commercially available locally (which has now been achieved), or until the Sponsor decided to discontinue the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Month 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects successfully completing study Infacort 003, whose inclusion criteria were:
- •Male and female children less than 6 years of age.
- •A diagnosis of adrenal insufficiency (AI) as confirmed by an inappropriately low cortisol usually with other supporting tests.
- •Receiving appropriate adrenocortical replacement therapy (hydrocortisone with/without fludrocortisone).
- •Adequately hydrated and nourished. In addition, the parents/carers must be able to understand and give written Informed Consent for this extension study.
排除标准
- •Clinically evident acute AI (adrenal crisis) (Note: the subject can be re-evaluated for eligibility once the episode is over)
- •Inability of the child to take oral therapy
- •Subjects with clinical signs of acute infection or fever on inclusion (Note: the subject can be re-evaluated for eligibility once the episode is over)
- •Any surgical or medical condition that in the opinion of the Investigator may place the subject at higher risk from his/her participation in the study
- •Parents/carers of subjects unwilling to consent to saving and propagation of pseudonymised medical data for study reasons
- •Subjects who are in a dependent relationship with the Investigator or the Sponsor
研究组 & 干预措施
Infacort
Infacort® granules
干预措施: Infacort® (Drug)
结局指标
主要结局
Incidence of Serious Adverse Events (SAEs) and Adverse Events (AEs)
时间窗: 29 months
The primary endpoint was the nature and occurrence of serious adverse events (SAEs) and adverse events (AEs) observed throughout the study. AEs were recorded from the time of the first intake of Infacort until the final visit.
次要结局
- Growth Velocity(29 months)
- Cortisol Levels(29 months)
- Number of Participants Exhibiting a Change in Tanner Development Stage(29 months)