A Phase 1 Pharmacokinetic Profiling Study in Patients Receiving Trientine Dihydrochloride for the Treatment of Wilson's Disease.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Pharmacokinetic measurements
研究概览
简要总结
To evaluate the effects of a single dose of trientine in children ≥6 years and adult patients with Wilson's disease.
详细描述
An open, single-dose study to determine the pharmacokinetics, and safety of oral administration of trientine dihydrochloride in both children and adult patients with Wilson's Disease. Blood samples will be taken pre-dose and at 10 time-points post-dose to investigate the pharmacokinetic profile of trientine dihydrochloride up to 12 hours after intake of study medication in 20 patients.
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
- 6 Years 至 90 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children ≥ 6 years and adult patients
- •Confirmed diagnosis of Wilson's disease by Leipzig-Score >3 (Ferenci et al 2003)
- •Current treatment with trientine dihydrochloride
- •Signed informed consent including parental consent in patients ≤ 18 years
- •Agree to remain in the study site1 for the PK measurements period.
排除标准
- •Known clinically significant allergy or hypersensitivity to drugs that, in the opinion of the investigator, may affect the patient's safety
- •Have any clinically significant conditions that would interfere with the collection or interpretation of the study results or would compromise the patient's health
- •Women of child bearing potential who do not use contraceptives, breastfeeding, or pregnant women
- •Severe anaemia (haemoglobin <9 mg/dL)
- •In the judgment of the Investigator, is likely to be noncompliant or uncooperative during the study, or unable to cooperate because of a language problem or poor mental development
- •Participation in any interventional clinical study at the same time or within the 4 weeks prior his study.
研究组 & 干预措施
Trientine dihydrochloride
干预措施: trientine dihydrochloride (Drug)
结局指标
主要结局
Pharmacokinetic measurements
时间窗: Evaluation of PK parameters will occur at 11 time points:Pre-dose), and at 30 minutes, 1, 1.5, 2, 3, 4, 5, 6, 8, and 12 hours post-dose
The main objective of the study is to evaluate the pharmacokinetics of a single dose of trientine dihydrochloride in children ≥6 years and adult patients with Wilson's disease by PK analysis. Non-compartmental or model-independent methods will be used to derive pharmacokinetic parameters using individual patient plasma concentration-time data. Pharmacokinetic parameters will include the Cmax, Tmax, T1/2, and the concentration-time curved (AUC0-t) and AUCinf.
次要结局
未报告次要终点