Condition category
Circulatory System
Date applied
04/02/2015
Date assigned
05/02/2015
Last edited
05/02/2015
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Background and study aims
Eisenmenger's Syndrome is a congenital condition affecting the heart which results in low oxygen levels in the blood and high blood pressure within the lungs. The increase in blood pressure is caused by thickening and narrowing of the blood vessels in the lung. Currently, treatment includes relatively new medications called “advanced therapies”, which are “pulmonary vasodilators”; this means that they open up the blood vessels and help blood flow through the lungs and improve the function of the heart. These medications have been shown to improve patients’ symptoms and also length of life. These treatments are expensive and are tightly regulated. At present, the Commissioners allow us to treat patients with up to two types of pulmonary vasodilators; normally both tablets. A third class of medication are the “prostanoids”. These are unstable medicines which need to be given regularly, either as a nebuliser (inhaled) or intravenously. At present, although licensed for use in Eisenmenger syndrome, we are not allowed to prescribe such medicines on top of two tablets. This study aims to investigate whether iloprost, an inhaled prostanoid, is beneficial in terms of exercise capacity in patients who are deteriorating or have an unsatisfactory response to two oral therapies.

Who can participate?
Adults (aged at least 18) with Eisenmenger's Syndrome.

What does the study involve?
Participants are randomly allocated into one of two groups. Those in group 1 are given iloprost . Those in group 2 are given an inhaled placebo. Each participant receives one study drug for 12 weeks, have a 1 week “washout” and then take the other medication for a further 12 weeks, in a so called “crossover” study. At the end of the study all participants are able to take iloprost indefinitely in an “openlabel” phase. During the study, participant responses such as walking distance, oxygen levels, quality of life questionnaire and heart function as determined by ultrasound (echocardiography) are monitored.

What are the possible benefits and risks of participating?
Not provided at time of registration

Where is the study run from?
Royal Brompton Hospital (UK)

When is the study starting and how long is it expected to run for?
December 2014 to May 2016

Who is funding the study?
Bayer PLC (UK)

Who is the main contact?
Ms Natalie Dormand

Trial website

Contact information

Type

Scientific

Primary contact

Ms Natalie Dormand

ORCID ID

Contact details

Royal Brompton Hospital
Sydney Street
London
SW3 6NP
United Kingdom

Additional identifiers

EudraCT number

2014-000091-25

ClinicalTrials.gov number

Protocol/serial number

18181

Study information

Scientific title

A single-centre, placebo-controlled, double-blinded, randomized, crossover study of Iloprost (Ventavis®) in patients with Eisenmenger syndrome

Acronym

Study hypothesis

This study aims to investigate whether iloprost, an inhaled prostanoid, is beneficial in terms of exercise capacity in Eisenmenger syndrome patients who are deteriorating or have an unsatisfactory response to two oral therapies.

Ethics approval

14/LO/1182

Study design

Randomised; Interventional; Design type: Treatment

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Other

Trial type

Treatment

Patient information sheet

Not available in web format, please use contact details to request a patient information sheet

Condition

Topic: Cardiovascular disease; Subtopic: Cardiovascular (all Subtopics); Disease: Congenital Heart Disease and Pulmonary Hypertension

Intervention

Use of nebulised Iloprost in addition to maximum oral pulmonary vasodilative therapy.

The study will involve patients receiving either iloprost or an inhaled placebo; both doctors and patients will be “blinded” to what they are receiving. The patient will receive one study drug for 12 weeks, have a 1 week “washout” and then take the other medication for a further 12 weeks, in a socalled “crossover” study. At the end of the study the patient will be able to take iloprost indefinitely in an “openlabel” phase. During the study we will monitor responses such as walking distance, oxygen levels, quality of life questionnaire and heart function as determined by ultrasound (echocardiography).

Intervention type

Other

Phase

Phase IV

Drug names

Primary outcome measures

Change in 6 Minute Walk Test over 3 months compared with placebo

Secondary outcome measures

N/A

Overall trial start date

15/12/2014

Overall trial end date

15/05/2016

Reason abandoned

Eligibility

Participant inclusion criteria

1. Patients > 18 years
2. Body weight > 40 kg
3. Functional class III (1998 WHO classification)
4. Patients with documented oxygen saturation < 90%, at rest or during exercise with room air
5. Patients with established pulmonary hypertension related to congenital heart disease and post – tricuspid lesion able to perform a 6minute
walk test with latest walking distance < 400m or deterioration of 30m within 1 Year on dual oral therapy or patient not tolerating oral therapy
6. Patients stable for at least 3 months prior to screening
7. Patients providing written informed consent

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

Planned Sample Size: 15; UK Sample Size: 15

Participant exclusion criteria

1. Pregnant patients
2. Trisomy 21
3. Obstructive lung disease (FEV1/FVC<60%)
4. Patients with systolic blood pressure <90 mm Hg
5. Patients with other conditions that may affect the ability to perform a six minute walk test
6. Patients unable to provide informed consent and comply with the protocol
7. Patients with known coronary artery disease
8. Patients who have started or stopped specific treatment for PAH within one month of screening, excluding anticoagulation
9. Patients active on an organ transplant list
10.Patients taking other investigational drugs/devices
11.Patients taking other Prostacyclin analogues, like Epoprostenol or Treprostinil
12.Patients with planned surgical intervention during the study period
13.Patients with HIV

Recruitment start date

15/12/2014

Recruitment end date

15/05/2016

Locations

Countries of recruitment

United Kingdom

Trial participating centre

Royal Brompton Hospital
Sydney Street
London
SW3 6NP
United Kingdom

Sponsor information

Organisation

Royal Brompton & Harefield NHS trust

Sponsor details

Royal Brompton Hospital
Sydney Street
London
SW3 6NP
United Kingdom

Sponsor type

Hospital/treatment centre

Website

Funders

Funder type

Government

Funder name

Bayer PLC (UK)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes