Revival of Autochthonous Adrenocortical Stem Cells in Autoimmune Addison's Disease
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 13
- 试验地点
- 1
- 主要终点
- Peak serum Cortisol following ACTH stimulation
研究概览
简要总结
Autoimmune Addison's disease (AAD) is a rare and debilitating disease in which an autoimmune attack progressively destroys the adrenal cortex. Untreated it is universally fatal and treated people are absolutely dependent upon steroid medications lifelong, with a consequent excess in morbidity and mortality. A key feature of the adrenal cortex is that its cells are responsive to changes in circulating adrenocorticotrophic hormone (ACTH) concentration. This study aims to regenerate adrenocortical steroidogenic cell function in patients with established autoimmune Addison's disease (AAD) by stimulating proliferation and differentiation of their progenitor cells, the adrenocortical stem cells (ACSCs) (1,2). Using daily subcutaneous ACTH, administered according to two different regimens over 20 weeks, we will investigate whether regeneration of adrenal steroidogenic function through revival of ACSC activity is a realistic possibility.
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
- 16 Years 至 66 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Established autoimmune adrenal failure for >1yr age 16 to 65
排除标准
- •Significant cardio-respiratory, chronic renal or non-autoimmune liver disease; malignancy
- •Asthma, current infectious disease, recent live vaccination, acute psychosis, peptic ulcer disease
- •Pregnancy, breast feeding or plan for pregnancy within 9 months
- •Known non-autoimmune cause for adrenal failure (haemorrhage, adrenoleukodystrophy etc.)
- •Known hypersensitivity or allergy to Synacthen
研究组 & 干预措施
Synacthen
active treatment
干预措施: depot tetracosactide (Drug)
结局指标
主要结局
Peak serum Cortisol following ACTH stimulation
时间窗: Tested at 20 weeks
次要结局
- Change in QoL(20 weeks)
研究者
SHS Pearce
Professor of Endocrinology
Newcastle University