Clinical Curative Effect Evaluation Study of Treatment of Oral Deferiprone Tablets in Aceruloplasminaemia Patients
试验速览
- 阶段
- 早期 1 期
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Change of glycated hemoglobin (HbA1c)
研究概览
简要总结
Aceruloplasminemia is an autosomal recessive disorder characterized by iron deposition in the brain and visceral organs. Deferiprone was used to treat aceruloplasminemia in previous study. In this study, we will assess the clinical curative effect evaluation of oral deferiprone in Chinese aceruloplasminemia patients.
详细描述
Diabetes is one of the most severe symptom in aceruloplasminemia. In this study, we use R2* of pancreas MRI to evaluate the effect of deferiprone treatment.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18-80 years
- •Diagnosed with aceruloplasminaemia
- •Genetically confirmed diagnosis of aceruloplasminaemia
排除标准
- •Contraindications to deferiprone therapy
- •Pregnancy was excluded in women of childbearing age
研究组 & 干预措施
Deferiprone treated
Deferiprone (25 mg/kg/day) was given to the enrolled patient.
干预措施: Deferiprone treated (Drug)
结局指标
主要结局
Change of glycated hemoglobin (HbA1c)
时间窗: Up to 3 months
HbA1c is a measure of long-term blood sugar control
Change of pancreas MRI
时间窗: Up to 3 months
Pancreas MRI is related to iron accumulation of pancreas
次要结局
未报告次要终点
研究者
Wan-Jin Chen
Professor
First Affiliated Hospital of Fujian Medical University