NCT02769624

Acute Effects of Inhaled Treprostinil in Fontan Patients

Terminated Phase 2 Results posted Last updated 29 July 2020

What this trial tests

Phase 2 trial testing Treprostinil in Congenital Heart Disease in 15 participants. Terminated before completion.

Timeline

21 February 2017

Primary endpoint
21 September 2018

21 September 2018

Quick facts

Lead sponsor	Children's Hospital Medical Center, Cincinnati
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	triple
Primary purpose	supportive care
Enrollment	15
Start date	21 February 2017
Primary completion	21 September 2018
Estimated completion	21 September 2018
Sites	1 location across United States

Drugs / interventions tested

• Treprostinil (TREPROSTINIL) — full drug profile →
• Placebo

Conditions studied

• Congenital Heart Disease — all drugs for Congenital Heart Disease →

Sponsor

Children's Hospital Medical Center, Cincinnati

Who can join

18 and older, any sex, with Congenital Heart Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Reduction in Liver Stiffness Through the Measurement of Shear Wave Ultrasound Elastography (m/s) Primary · At rest and following dose 2

At rest and then following maximal exercise, shear wave ultrasound elastography will be performed to assess if the study medication shows an improvement in liver stiffness (reduction in m/s number) following maximal exercise versus at rest.

Rest

Group	Value	95% CI
Treprostinil	2.39	± 0.67
Placebo	2.38	± 0.71

Following maximal exercise

Group	Value	95% CI
Treprostinil	2.79	± 0.54
Placebo	2.89	± 0.51

Venous Pressure (mmHg) at Rest and Peak Exercise Secondary · At rest and at peak incremental exercise

Venous pressure was measured both at rest and during an incremental ramp exercise test. Comparison was made to evaluate if the treprostinil study medication had a different effect in venous pressure measurements during peak exercise versus placebo.

Rest

Group	Value	95% CI
Treprostinil	12.8	± 3.08
Placebo	12.1	± 2.43

Peak Incremental Exercise

Group	Value	95% CI
Treprostinil	22.8	± 6.16
Placebo	22.6	± 8.04

Exercise Endurance Time (in Minutes) Secondary · Immediately following dose 3

A constant work rate exercise test will be performed to determine exercise endurance

Group	Value	95% CI
Treprostinil	5.19	± 0.89
Placebo	5.82	± 1.23

Peak Volume of Oxygen Consumption (VO2) (ml/kg/Min) Secondary · Maximal exercise test (5-10 minutes)

Measuring the difference in peak/maximal VO2 during incremental exercise testing to assess if the response during peak exercise is different when utilizing treprostinil versus a placebo.

Group	Value	95% CI
Treprostinil	23.4	± 6.67
Placebo	24.6	± 9.04

Minute Volume and Carbon Dioxide Volume Slope (VE/VCO2) During Incremental Exercise Secondary · Maximal exercise test (5-10 minutes)

Measuring the difference in peak/maximal VE/VCO2 during incremental exercise testing to assess if the response during peak exercise is different when utilizing treprostinil versus a placebo. The VE/VCO2 slope is used to show the relationship between minute ventilation and carbon dioxide production.

Group	Value	95% CI
Treprostinil	26.8	± 4.95
Placebo	26.1	± 6.35

VO2 (ml/kg/Min) at Anaerobic Threshold Secondary · Maximal exercise test (5-10 minutes)

Measuring the difference in VO2 at anaerobic threshold of an incremental exercise test to assess the variance in response with treprostinil versus placebo

Group	Value	95% CI
Treprostinil	17.3	± 3.63
Placebo	18.1	± 5.36

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were assessed and recorded following signing of informed consent through two weeks following the last study visit (period ranging from 6-9 weeks).. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treprostinil

Serious: 0/14 (0%)

Deaths: 0/14

Placebo

Serious: 0/15 (0%)

Deaths: 0/15

Other adverse events (10 terms — click to expand)

Reaction	System	Treprostinil	Placebo
Cough	Respiratory, thoracic and mediastinal disorders	—	—
Nausea	Gastrointestinal disorders	—	—
Sore Throat	Respiratory, thoracic and mediastinal disorders	—	—
Chest Tightness	Respiratory, thoracic and mediastinal disorders	—	—
Dizziness	Ear and labyrinth disorders	—	—
Increase in Liver Lab Values	Hepatobiliary disorders	—	—
Abnormal ECG Rhythm	Cardiac disorders	—	—
Headache	Musculoskeletal and connective tissue disorders	—	—
Weakness	General disorders	—	—
Lowered blood pressure	Cardiac disorders	—	—

Data from ClinicalTrials.gov NCT02769624 adverse events section.

Sponsor's own description

In this pilot study investigators propose to explore the effects of a locally delivered inhaled pulmonary vasodilator (inhaled treprostinil) on exercise performance, pulmonary blood flow, venous pressure response and vascular function in stable Fontan patients. Investigators will also assess the effects of resting and acute rises in exercise induced systemic venous pressure on liver stiffness and will also assess whether treprostinil will attenuate the acute stiffness increase that investigators expect to see. This will be a prospective, randomized, double-blinded placebo controlled, crossover trial. Following recruitment and informed consent, each participant will undergo three study visits, including baseline testing (visit 1), followed by two sets of exercise and vascular function tests (visit 2 and visit 3) at CCHMC. Twenty-six patients will be enrolled in this study.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.