Midodrine in Hepatopulmonary Syndrome

Mayo Clinic

Status and phase: Completed; Phase 1

Conditions: Hepatopulmonary Syndrome (HPS)

Treatments: Drug: Midodrine

Study type: Interventional

Funder types: Other

Identifiers

NCT03600870

17-006221

Details and patient eligibility

About

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.

Enrollment: 10 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Moderate to very severe hepatopulmonary syndrome, defined as the presence of all of the following:

  1. Liver disease or portal hypertension
  2. Intrapulmonary shunting on contrast-enhanced echocardiogram
  3. 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Open Label

Experimental group

Description:

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.

Treatment:

Drug: Midodrine