Imaging and Biomarker Evaluation of Hepatic Stiffness in Children Enrolled in the Fontan Udenafil Exercise Longitudinal Study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 88
- 试验地点
- 17
- 主要终点
- Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Ultrasound Shear Wave Elastography
研究概览
简要总结
A study to evaluate the efficacy of MZ101 therapy in reducing liver stiffness.
详细描述
This is open-label, long-term prospective study in a cohort of Fontan subjects taking drug daily for one year. The study will determine the scope of hepatic stiffness in the cohort by ultrasound and magnetic resonance imaging elastography and evaluate MZ101 pharmacotherapy as a potentially efficacious treatment to reduce liver stiffness.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Enrollment in on-going Phase 3 Open-Label Safety Study
- •Informed assent from subject, informed consent from parent/legal guardian as appropriate
排除标准
- •Non-enrollment in the on-going Phase 3 Open-Label Study
- •Subjects with contra-indications for MRI (these subjects will be excluded from the MRI component of this study)
- •Other exclusionary criteria will match those used for the Open-Label Safety Study
研究组 & 干预措施
Udenafil Continuation (U+)
Subjects who were receiving Udenafil 87.5 mg BID in FUEL study prior to enrolling to this study (FALD).
干预措施: Udenafil (Drug)
Udenafil Naive (U-)
Subjects who were receiving Placebo BID in FUEL study prior to enrolling to this study (FALD).
干预措施: Udenafil (Drug)
结局指标
主要结局
Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Ultrasound Shear Wave Elastography
时间窗: 52 weeks
Liver stiffness was measured at baseline and 52 weeks using Ultrasound Shear Wave Elastography (USWE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness.
Effect of Udenafil 87.5 mg BID Treatment on Liver Stiffness in Udenafil Continuation and Naive Groups After 52 Weeks, as Determined by Magnetic Resonance Elastography
时间窗: 52 weeks
Liver stiffness was measured at baseline and 52 weeks using Magnetic Resonance Elastography (MRE) separately in Udenafil Continuation (U+) and Udenafil Naive (U-) groups. A decrease in value indicates a decrease in liver stiffness.
次要结局
- Effect of Udenafil 87.5 mg BID on Enhanced Liver Fibrosis (ELF) Score After 52 Weeks of Treatment(52 weeks)
- Effect of Udenafil 87.5 mg BID on Brain Natriuretic Peptide After 52 Weeks of Treatment(52 weeks)