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NCT Number: NCT03600870

Midodrine in Hepatopulmonary Syndrome

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.

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Key information

Conditions

Hepatopulmonary Syndrome (HPS) Digestive System Diseases Hepatopulmonary Syndrome Liver Diseases Lung Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Midodrine

Drug

Midodrine is an oral alpha-1 agonist that increases vascular tone. It is administered orally. 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.

Primary outcomes

  1. Safety and tolerability (adverse events (AEs))

    Time frame: 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.

Secondary outcomes

  1. Arterial Oxygenation

    Time frame: 3 months and 6 months

    Describe the effect of midodrine on:

    • arterial oxygenation (PaO2 and A-a gradient )(mmHg)

  2. Diffusion capacity

    Time frame: 3 months and 6 months

    Describe the effect of midodrine on:

    • percent predicted diffusion capacity for carbon monoxide (Range 0-100%)

  3. Cardiac output

    Time frame: 3 months and 6 months

    Describe the effect of midodrine on cardiac output (L/min) as estimated by echocardiogram in patients with HPS.

  4. Intrapulmonary shunting

    Time frame: 3 months and 6 months

    Describe the effect of midodrine on severity (mild, moderate or severe) of intrapulmonary shunting (as assessed by echocardiogram shunt study) in patients with HPS.

  5. Intrapulmonary shunting

    Time frame: 6 months

    Describe the effect of midodrine on severity of intrapulmonary shunting (as assessed by percent shunt index (0-100%) on technetium macroaggregated albumin scan) in patients with HPS.

  6. Symptoms as assessed by Modified Medical Research Council (MMRC)dyspnea scale

    Time frame: 3 months and 6 months

    Describe the effect of midodrine on MMRC dyspnea scale (0-4). Higher numbers indicate more severe dyspnea.

  7. 6 minute walk distance

    Time frame: 3 months and 6 months

    Describe the effect of midodrine on 6 minute walk distance, in meters.

Sponsors and collaborators

Lead sponsor

Mayo Clinic

Other

Registry information

Official study title

A Phase 1 Proof-of-concept Clinical Trial Evaluating the Safety and Tolerability of Midodrine in Hepatopulmonary Syndrome

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Jul 26, 2018
Registry last updated
Jan 6, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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