- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03600870
Midodrine in Hepatopulmonary Syndrome
January 5, 2021 updated by: Hilary M. DuBrock,, Mayo Clinic
A Phase 1 Proof-of-concept Clinical Trial Evaluating the Safety and Tolerability of 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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
10
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Minnesota
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Rochester, Minnesota, United States, 55905
- Mayo Clinic in Rochester
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: 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.
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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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Safety and tolerability (adverse events (AEs))
Time Frame: 6 months
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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.
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6 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Arterial Oxygenation
Time Frame: 3 months and 6 months
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Describe the effect of midodrine on: • arterial oxygenation (PaO2 and A-a gradient )(mmHg) |
3 months and 6 months
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Diffusion capacity
Time Frame: 3 months and 6 months
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Describe the effect of midodrine on: • percent predicted diffusion capacity for carbon monoxide (Range 0-100%) |
3 months and 6 months
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Cardiac output
Time Frame: 3 months and 6 months
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Describe the effect of midodrine on cardiac output (L/min) as estimated by echocardiogram in patients with HPS.
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3 months and 6 months
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Intrapulmonary shunting
Time Frame: 3 months and 6 months
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Describe the effect of midodrine on severity (mild, moderate or severe) of intrapulmonary shunting (as assessed by echocardiogram shunt study) in patients with HPS.
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3 months and 6 months
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Intrapulmonary shunting
Time Frame: 6 months
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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.
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6 months
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Symptoms as assessed by Modified Medical Research Council (MMRC)dyspnea scale
Time Frame: 3 months and 6 months
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Describe the effect of midodrine on MMRC dyspnea scale (0-4).
Higher numbers indicate more severe dyspnea.
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3 months and 6 months
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6 minute walk distance
Time Frame: 3 months and 6 months
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Describe the effect of midodrine on 6 minute walk distance, in meters.
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3 months and 6 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Hilary M DuBrock, M.D., Mayo Clinic
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
August 2, 2018
Primary Completion (ACTUAL)
December 1, 2020
Study Completion (ACTUAL)
December 1, 2020
Study Registration Dates
First Submitted
June 20, 2018
First Submitted That Met QC Criteria
July 25, 2018
First Posted (ACTUAL)
July 26, 2018
Study Record Updates
Last Update Posted (ACTUAL)
January 6, 2021
Last Update Submitted That Met QC Criteria
January 5, 2021
Last Verified
January 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Respiratory Tract Diseases
- Lung Diseases
- Disease
- Liver Diseases
- Syndrome
- Hepatopulmonary Syndrome
- Physiological Effects of Drugs
- Adrenergic Agents
- Neurotransmitter Agents
- Molecular Mechanisms of Pharmacological Action
- Autonomic Agents
- Peripheral Nervous System Agents
- Adrenergic alpha-Agonists
- Adrenergic Agonists
- Sympathomimetics
- Vasoconstrictor Agents
- Adrenergic alpha-1 Receptor Agonists
- Midodrine
Other Study ID Numbers
- 17-006221
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hepatopulmonary Syndrome (HPS)
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Sun JieRecruitingHepatopulmonary Syndrome (HPS)China
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University of Michigan Rogel Cancer CenterCompletedHemophagocytic Syndrome (HPS)United States
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Unity Health TorontoRecruitingHepatopulmonary SyndromeCanada
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University of PennsylvaniaNational Heart, Lung, and Blood Institute (NHLBI)Terminated
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Unity Health TorontoUniversity of TorontoCompleted
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University of Alabama at BirminghamTerminated
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Clinical Trials on Midodrine
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University of VirginiaWithdrawn
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China National Center for Cardiovascular DiseasesFirst Affiliated Hospital, Sun Yat-Sen University; RenJi Hospital; First Affiliated... and other collaboratorsRecruiting
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University of AlbertaNovartisCompletedRefractory Ascites | Type 2 Hepatorenal SyndromeCanada
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James J. Peters Veterans Affairs Medical CenterCompletedHypothermia | Mild Cognitive Impairment | TetraplegiaUnited States
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James J. Peters Veterans Affairs Medical CenterThe Craig H. Neilsen FoundationActive, not recruitingHypothermia | Mild Cognitive Impairment | TetraplegiaUnited States
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Icahn School of Medicine at Mount SinaiCompleted
-
Benha UniversityNew Jeddah Clinic HospitalCompleted
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Seoul National University HospitalCompletedOrthostatic; Hypotension, NeurogenicKorea, Republic of
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National Center for Research Resources (NCRR)Roberts PharmaceuticalCompletedOrthostatic HypotensionUnited States