Midodrine in Hepatopulmonary Syndrome

NCT03600870 | Completed Phase 1 FDA Drug

• 1 other identifier: 17-006221
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Hepatopulmonary Syndrome (HPS)

Outcome Measures

Primary Outcomes (1)

• 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.

  6 months

Secondary Outcomes (7)

• Arterial Oxygenation

  3 months and 6 months

• Diffusion capacity

  3 months and 6 months

• Cardiac output

  3 months and 6 months

• Intrapulmonary shunting

  3 months and 6 months

• Intrapulmonary shunting

  6 months

Study Arms (1)

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.

Drug: Midodrine

Interventions

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.

Open Label

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Hepatopulmonary Syndrome

Interventions

Midodrine

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines

Study Officials

• Hilary M DuBrock, M.D.

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: June 20, 2018
• First Posted: July 26, 2018
• Study Start: August 2, 2018
• Primary Completion: December 1, 2020
• Study Completion: December 1, 2020
• Last Updated: January 6, 2021

Record last verified: 2021-01

Locations

US (1)