A Phase 3 Open-label Study of Infacort® in Neonates, Infants and Children Less Than 6 Years of Age With Adrenal Insufficiency
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Serum Cortisol Concentration up to 240 Minutes
研究概览
简要总结
The study will be conducted in a total of 24 subjects before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism.
The study will consist of three consecutive cohorts. Cohort 1 will include 12 subjects aged between 2 and < 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to <2 years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only if again no safety concerns emerge, then 6 neonates aged from birth to <28 days will be enrolled (Cohort 3).
The decision to continue after each cohort will be based on the recommendation of an Independent Data Monitoring Committee (IDMC).
详细描述
This is a Phase 3, open label, single centre study of Infacort® in neonates, infants and children less than 6 years of age with adrenal insufficiency. The study will be conducted in a total of 24 subjects, before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism. Due to the rare occurrence of adrenal insufficiency it is expected that for the third cohort of this study (neonates) only subjects with CAH will be recruited.
The study will consist of three consecutive parts. Cohort 1 will include 12 subjects aged between 2 and < 6 years. If no safety concerns emerge, then 6 subjects aged 28 days to <2 years will be enrolled (Cohort 2). A review of accumulated data will be undertaken and only if again no safety concerns emerge, then 6 neonates aged from birth to <28 days will be enrolled (Cohort 3).
The decision to continue after each cohort will be based on the recommendation of an Independent Data Monitoring Committee (IDMC).
The study will consist of a screening visit (Visit 1 performed as a minimum the day before the intake of study drug), one treatment visit (Visit 2, Day 1), a follow-up visit (Visit 3) one to three days after intake of study drug (Day 2, Day 3 or Day 4) and a follow-up telephone call (Visit 4) 7 - 10 days after intake of study drug. Study completion evaluation will be performed at Visit 3. Parents/ carers will have at least 1 night to consider participation of their child before completing written informed consent. Children aged 3 - 6 years will be informed about their involvement in the study in the presence of their parents/carers.
All subjects will receive their standard treatment including fludrocortisone other than the dose of hydrocortisone that is to be substituted by Infacort®.
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 Week 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female children less than 6 years of age.
- •A diagnosis of adrenal insufficiency 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.
- •Ability of parents/carers to understand and give written Informed Consent
排除标准
- •Clinically evident acute adrenal insufficiency (adrenal crisis).
- •Inability of the child to take oral therapy.
- •Concomitant therapy (other than that required to treat adrenal insufficiency, Vitamin D, Fluoride, Thyroxine and growth hormone).
- •Subjects with clinical signs of acute infection or fever on Day
- •Any surgical or medical condition which 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 dependent on the investigator or the sponsor.
研究组 & 干预措施
Infacort
Infacort® is a dry granule formulation of hydrocortisone stored in capsules that will be available in different strengths (0.5, 1.0, 2.0 and 5.0mg).
The clinically-appropriate dose, based on standard individualised treatment, will be administered, given as a single dose orally. This will usually be equivalent to the previous day's dose.
干预措施: Infacort® (Drug)
结局指标
主要结局
Serum Cortisol Concentration up to 240 Minutes
时间窗: 240 minutes
The primary endpoint will be the maximum levels of serum cortisol concentration up to 240 minutes after intake of study drug as determined by the central laboratory.
次要结局
- Serum Cortisol Concentration up to 6 Hours(240 minutes)
- Subject Assessment of Taste of the Product(1 minute)
- Incidence of Serious Adverse Events (SAEs) and Adverse Events (AE)(7-10 days)