A Phase 1 Proof-of-concept Clinical Trial Evaluating the Safety and Tolerability of Midodrine in Hepatopulmonary Syndrome
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Safety and tolerability (adverse events (AEs))
研究概览
简要总结
This proof-of-concept clinical trial will determine the safety and tolerability of midodrine in patients with hepatopulmonary syndrome (HPS). Exploratory endpoints will assess the effect of midodrine on oxygenation, intrapulmonary shunting and symptoms.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Moderate to very severe hepatopulmonary syndrome, defined as the presence of all of the following:
- •Liver disease or portal hypertension
- •Intrapulmonary shunting on contrast-enhanced echocardiogram
- •Hypoxemia [A-a gradient ≥15mmHg (or ≥20mmHg if age >64) and PaO2<80mmHg on arterial blood gas testing]
- •Ability to provide informed consent
- •Ability to comply with study medication use and testing, in the opinion of the principal investigator or co-investigator
排除标准
- •Vulnerable study population, including imprisoned individuals, non-English speaking patients
- •Participation in other investigational drug studies
- •Any of the following conditions:
- •Systolic blood pressure>160mmHg or diastolic blood pressure >100mmHg
- •Heart rate <50bpm
- •Urinary retention at baseline
- •Left ventricular ejection fraction <50%
- •Women who are pregnant, nursing, or who plan to become pregnant while in the trial
- •Women of child-bearing potential not willing or able to use highly effective methods of birth control
研究组 & 干预措施
Open Label
All subjects enrolled will be assigned to receive midodrine. The initial starting dose will be midodrine 5mg orally three times a day. After 7-10 days, patients will increase the dose to 10mg three times a day as tolerated if no adverse effects occur. Treatment duration will be 6 months.
干预措施: Midodrine (Drug)
结局指标
主要结局
Safety and tolerability (adverse events (AEs))
时间窗: 6 months
Outcome will be defined by the incidence of adverse events (AEs) that occur during the study period. An adverse event is defined as any symptom, sign, illness or experience that develops or worsens in severity during the course of the study. Abnormal results of diagnostic procedures are considered to be AEs if the abnormality results in study withdrawal, is associated with a serious AE, is associated with clinical signs or symptoms, leads to additional treatment or further diagnostic tests or is considered by the investigator to be of clinical significance. Each adverse event will be further characterized by severity and relationship to the study drug. Adverse event are classified as serious if they are: fatal, life-threatening, require or prolongs hospital stay, lead to persistent or significant disability or incapacity or a congenital anomaly or birth defect.
次要结局
- Cardiac output(3 months and 6 months)
- Arterial Oxygenation(3 months and 6 months)
- Diffusion capacity(3 months and 6 months)
- Intrapulmonary shunting(6 months)
- Symptoms as assessed by Modified Medical Research Council (MMRC)dyspnea scale(3 months and 6 months)
- 6 minute walk distance(3 months and 6 months)
研究者
Hilary M. DuBrock,
Assistant Professor
Mayo Clinic