Phase 2
N=16
Ambrisentan in Single Ventricle
Hypoplastic Left Heart Syndrome · Hypoplastic Right-sided Heart Complex
Bottom Line
View on ClinicalTrials.gov: NCT02080637 ↗Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Sep 2019
Primary outcome: Primary: Area Under the Curve for Ambrisentan Plasma Concentration — 7871.46 ng*hr/mL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ambrisentan (Drug); Placebo (Other)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Kevin Hill
- Primary completion
- Jul 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Area Under the Curve for Ambrisentan Plasma Concentration |
7871.46 | — |
| PRIMARY Change in Pulmonary Vascular Resistance Index |
-0.5; -0.2 | 0.01 sig |
| SECONDARY Amount of Chest Tube Drainage Post Fontan Operation |
1325; 1356 | 0.94 |
| SECONDARY Duration of Chest Tube Drainage Post Fontan Operation |
6.54; 5.33 | 0.61 |
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.
Eligibility Criteria
Inclusion Criteria
- Age ≥ 24 months; ≤120 months.
- History of congenital heart disease with severe hypoplasia of a right or left ventricle.
- Undergoing Fontan surgery as part of standard clinical care.
- Availability and willingness of the parent/legally authorized representative to provide written informed consent and, as appropriate, assent from the child.
Exclusion Criteria
- History of serious adverse event related to ambrisentan administration.
- History of ambrisentan exposure within 48 hours of the study.
- Presence of pulmonary venous obstruction.
- Treatment with cyclosporin.
- Any of the following - as determined by the attending physician
- Significant hemodynamic instability
- Sepsis.
- Need for ECMO support.
- Renal failure defined as serum creatinine > 2 times higher than the upper limit of normal.
- Liver dysfunction defined as alanine aminotransferase or aspartate aminotransferase > 3 times higher than the upper limit of normal.
- Thrombocytopenia defined as a platelet count 12mm Hg).
Data sourced from ClinicalTrials.gov (NCT02080637). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.