IMProving DRug Dosing and Outcomes for Single VEntricle Patients With Fontan Associated Liver Disease
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Pravastatin concentration as measured by area under the curve (AUC)
研究概览
简要总结
This is a single center, open-label, prospective, investigation to quantify the effects liver congestion and fibrosis has on hepatic drug metabolism and transport in children, adolescents, and young adults with Fontan circulation.
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
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •> 8 years
- •Status Post Fontan Completion
- •Ability to provide informed permission-assent (<18 years) or consent (≥18 years)
- •Fasting overnight (~8 hours)
排除标准
- •Pregnancy
- •Non-fasting
- •Non-removable metal in body or where magnetic resonance imaging (MRI) is considered unsafe
- •Sildenafil and/or Pravastatin therapy within last 2 months
- •History of underlying or laboratory evidence of underlying intestinal, metabolic, autoimmune, renal disease that can alter Sildenafil and Pravastatin disposition (absorption, metabolism, distribution, or clearance)
- •Pharmacotherapy that interacts with Sildenafil (cytochrome P450 3A4 and P450 3A5 (CYP3A4/5) inducers/inhibitors) and/or Pravastatin (organic anion transporting polypeptide (OATP1B1) inducers/inhibitors)
- •Inability to swallow a tablet
- •>5X the age-specific upper limit of normal for aspartate aminotransferase (AST) and alanine aminotransferase (ALT), total and conjugated bilirubin
- •Diarrhea in the last 24 hours *History of solid organ transplantation
研究组 & 干预措施
Assessment Arm
Assessment group will receive a single dose of oral sildenafil and oral pravastatin.
干预措施: Sildenafil 10mg oral tablet (participants <20kg), or 20mg oral tablet (participants ≥20kg) (Drug)
Assessment Arm
Assessment group will receive a single dose of oral sildenafil and oral pravastatin.
干预措施: Pravastatin 20 mg oral tablet (ages <13 years), or 40 mg oral tablet (≥14 years) (Drug)
结局指标
主要结局
Pravastatin concentration as measured by area under the curve (AUC)
时间窗: 2 years
Area under the time-exposure curve (AUC 0-n) for pravastatin as determinants of the dose-exposure relationship.
Sildenafil concentration as measured by area under the curve (AUC)
时间窗: 2 years
Area under the time-exposure curve (AUC 0-n) for sildenafil as determinants of the dose-exposure relationship.
次要结局
未报告次要终点
研究者
Jon Wagner
Director, Division of Clinical Pharmacology, Toxicology, and Therapeutic Innovation
Children's Mercy Hospital Kansas City