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Ambrisentan in Single Ventricle

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ClinicalTrials.gov Identifier: NCT02080637
Recruitment Status : Recruiting
First Posted : March 6, 2014
Last Update Posted : May 3, 2017
Information provided by (Responsible Party):
Kevin Hill, Duke University Medical Center

Brief Summary:

Purpose: To evaluate the pharmacokinetics, bioavailability and hemodynamic efficacy of ambrisentan after Fontan surgical palliation of single ventricle heart defects.

Study activities and population group: Children undergoing Fontan surgical palliation for single ventricle defects will be eligible for the study. Up to 20 subjects will be enrolled (16 ambrisentan, 4 placebo) and will receive 3 days (3 doses) of ambrisentan starting on post-operative day #1 upon returning from the operating room. Ambrisentan plasma levels will be obtained at specified time points during treatment. Post-operative monitoring lines will be used to measure effects of ambrisentan on hemodynamics and pulmonary / systemic endothelial function.

Condition or disease Intervention/treatment Phase
Hypoplastic Left Heart Syndrome Hypoplastic Right-sided Heart Complex Drug: Ambrisentan Other: Placebo Phase 2

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: Safety, Pharmacokinetics (PK) and Hemodynamic Effects of Ambrisentan in Single Ventricle Pediatric Patients
Study Start Date : July 2015
Estimated Primary Completion Date : June 2018
Estimated Study Completion Date : July 2018

Arm Intervention/treatment
Active Comparator: Ambrisentan
Oral ambrisentan 2.5 - 5 mg, single dose, once daily
Drug: Ambrisentan
Once daily oral dosing
Other Name: Leitaris
Placebo Comparator: Placebo
Oral placebo 2.5 - 5 mg, single dose, once daily
Other: Placebo

Primary Outcome Measures :
  1. Plasma concentration of Ambrisentan [ Time Frame: Plasma samples collected at 0-1,1-6,18-30 and 40-60hrs after administration of the first ambrisentan dose. ]
    Change Plasma concentration of ambrisentan

Secondary Outcome Measures :
  1. Amount and duration of chest tube drainage post Fontan Operation [ Time Frame: Chest tube output will be measured daily for the duration of the post-operative hospitalization or for 30 days whichever is shorter. Chest tube duration will be calculated as the number of days from placement to removal. ]
    Collect these data to assess whether ambrisentan improves post-operative outcomes.

  2. Change in hemodynamic parameters [ Time Frame: Hemodynamic evaluation performed prior to initiation of study drug and at 0-1,1-6,18-30 and 40-60hrs after administration of the first ambrisentan dose ]
    Hemodynamic data, including Fontan pressures and saturations, will be collected at the specified timepoints

  3. Safety [ Time Frame: 30 days after last ambrisentan dose ]
    Adverse events will be collected during hospitalization and for 30 days after administration of the last ambrisentan dose

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Ages Eligible for Study:   24 Months to 120 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Age ≥ 24 months; ≤120 months.
  2. History of congenital heart disease with severe hypoplasia of a right or left ventricle.
  3. Undergoing Fontan surgery as part of standard clinical care.
  4. Availability and willingness of the parent/legally authorized representative to provide written informed consent and, as appropriate, assent from the child.

Exclusion Criteria:

  1. History of serious adverse event related to ambrisentan administration.
  2. History of ambrisentan exposure within 48 hours of the study.
  3. Presence of pulmonary venous obstruction.
  4. Treatment with cyclosporin.
  5. Any of the following - as determined by the attending physician

    • Significant hemodynamic instability
    • Sepsis.
    • Need for ECMO support.
  6. Renal failure defined as serum creatinine > 2 times higher than the upper limit of normal.
  7. Liver dysfunction defined as alanine aminotransferase or aspartate aminotransferase > 3 times higher than the upper limit of normal.
  8. Thrombocytopenia defined as a platelet count < 50 000 cells/µL.
  9. Leukopenia defined as white blood cells < 2500 cells/µL.
  10. Anemia defined as hemoglobin < 8mg/dL.
  11. Atrial hypertension (mean LA pressure > 12mm Hg).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02080637

Contact: Kevin Hill, MD 919-681-2880 kevin.hill@dm.duke.edu
Contact: Mingfen Xu, MSN 919-668-6352 mingfen.xu@duke.edu

United States, North Carolina
Duke Universtiy Hospital Recruiting
Durham, North Carolina, United States, 27710
Contact: Mingfen Xu, MSN    919-668-6352    mingfen.xu@duke.edu   
Sponsors and Collaborators
Kevin Hill
Principal Investigator: Kevin Hill, MD Duke University

Responsible Party: Kevin Hill, Assist Professor, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT02080637     History of Changes
Other Study ID Numbers: Pro00050118
First Posted: March 6, 2014    Key Record Dates
Last Update Posted: May 3, 2017
Last Verified: May 2017

Keywords provided by Kevin Hill, Duke University Medical Center:
Single ventricle

Additional relevant MeSH terms:
Hypoplastic Left Heart Syndrome
Cleft Palate
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Situs Inversus
Jaw Abnormalities
Jaw Diseases
Musculoskeletal Diseases
Maxillofacial Abnormalities
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Stomatognathic Diseases
Mouth Abnormalities
Mouth Diseases
Stomatognathic System Abnormalities
Malformations of Cortical Development, Group I
Malformations of Cortical Development
Nervous System Malformations
Nervous System Diseases
Antihypertensive Agents