Hepatopulmonary Syndrome (HPS)Phase 1NCT03600870
Midodrine in Hepatopulmonary Syndrome
A Phase 1 study testing Midodrine in people with Hepatopulmonary Syndrome (HPS), run by Mayo Clinic at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatopulmonary Syndrome (HPS)
- What is being tested?
- Midodrine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Aug 2018to 1 Dec 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: 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.
- Conditions
- Hepatopulmonary Syndrome (HPS)
- Being tested
- Midodrine
- Medical subject headings
- Hepatopulmonary Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MidodrineDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Open Label | Experimental | 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. Receives: Drug: Midodrine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability (adverse events (AEs))
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.
Measured: 6 months
Secondary outcomes (7)
Arterial Oxygenation
Describe the effect of midodrine on:
- arterial oxygenation (PaO2 and A-a gradient )(mmHg)
Measured: 3 months and 6 months
Diffusion capacity
Describe the effect of midodrine on:
- percent predicted diffusion capacity for carbon monoxide (Range 0-100%)
Measured: 3 months and 6 months
Cardiac output
Describe the effect of midodrine on cardiac output (L/min) as estimated by echocardiogram in patients with HPS.
Measured: 3 months and 6 months
Intrapulmonary shunting
Describe the effect of midodrine on severity (mild, moderate or severe) of intrapulmonary shunting (as assessed by echocardiogram shunt study) in patients with HPS.
Measured: 3 months and 6 months
Intrapulmonary shunting
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.
Measured: 6 months
Symptoms as assessed by Modified Medical Research Council (MMRC)dyspnea scale
Describe the effect of midodrine on MMRC dyspnea scale (0-4). Higher numbers indicate more severe dyspnea.
Measured: 3 months and 6 months
6 minute walk distance
Describe the effect of midodrine on 6 minute walk distance, in meters.
Measured: 3 months and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2018
- Study start2 Aug 2018
- Primary completion1 Dec 2020
- Study completion1 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatopulmonary Syndrome (HPS)Studies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hilary M. DuBrock,, Mayo Clinic
- Investigators
- Hilary M DuBrock, M.D., Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03600870
- Sponsor's protocol ID
- 17-006221
- Status last verified
- Jan 2021
- First submitted
- 20 Jun 2018
- Last updated
- 6 Jan 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Proof-of-concept Clinical Trial Evaluating the Safety and Tolerability of Midodrine in Hepatopulmonary Syndrome
- ClinicalTrials.gov ID
- NCT03600870
- Acronym / sponsor ID
- 17-006221
- Conditions
- Hepatopulmonary Syndrome (HPS)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 2 Aug 2018 (actual)
- Primary completion
- 1 Dec 2020 (actual)
- Study completion
- 1 Dec 2020 (actual)
- First posted
- 26 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open Label | Experimental | Drug: MidodrineAll 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Moderate to very severe hepatopulmonary syndrome, defined as the presence of all of the following:
- Ability to provide informed consent
- Ability to comply with study medication use and testing, in the opinion of the principal investigator or co-investigator
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MidodrineMidodrine 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. | Drug | Open Label |
Primary outcome
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.
7 secondary outcomes
Arterial Oxygenation
Time frame: 3 months and 6 months
Diffusion capacity
Time frame: 3 months and 6 months
Cardiac output
Time frame: 3 months and 6 months
Intrapulmonary shunting
Time frame: 3 months and 6 months
Intrapulmonary shunting
Time frame: 6 months
Symptoms as assessed by Modified Medical Research Council (MMRC)dyspnea scale
Time frame: 3 months and 6 months
6 minute walk distance
Time frame: 3 months and 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2021.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hilary M DuBrock, M.D. principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Midodrine in people with Hepatopulmonary Syndrome (HPS), run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability (adverse events (AEs)) (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jan 2021.