HP 3.2 Bosentan therapy for pulmonary arterial hypertension (completed)

This is a 24-week, open-label, uncontrolled, prospective, multi-center treatment study involving 60 patients aged 14 and over who show clinical signs of Eisenmenger’s syndrome (cyanosis due to an intra- or extracardial shunt in connection with pulmonary arterial hypertension).

The table provides a general overview of the medical procedures that form part of the planned bosentan therapy.

A thorough basic medical examination is carried out during an inpatient stay. In addition to heart catheterisation and tests to determine pulmonary vascular resistance, it includes an MRI heart scan, various tests of exercise tolerance and ECGs. This inpatient stay takes place at the beginning of treatment and again after 24 weeks of treatment. Treatment is started during the first inpatient stay.

Outpatient visits are scheduled during the intervening time between the two “major” examinations. These generally involve a heart check-up consisting of all the necessary medical procedures (physical examination, heart ultrasound, exercise tolerance). During certain of these outpatient visits, blood samples will also be taken and forwarded to the laboratory for complementary tests (verification/comparison with initial parameters).

Lastly, a telephone consultation will be conducted to ask the patient about the efficacy and side effects of the treatment on a date that coincides with a visit to the patient’s GP to obtain blood samples for the monitoring of heart functions. If this visit is not possible, an outpatient visit must be conducted instead.

Important: Attempts to start a pregnancy must be avoided prior to and during the treatment, because the effects of the drug bosentan on the unborn child are not entirely elucidated. If any women of childbearing age participate in the study, a pregnancy test will be conducted prior to the commencement of treatment and again after 24 weeks of treatment, using a blood sample taken simultaneously with the standard laboratory tests.

All patients accepted for treatment will be prescribed increasing doses of bosentan. For the first 4 weeks, the test medication will be administered at 50% of the planned final dose. From the 4th week onwards, successively higher doses will be administered until the final level is reached, after which the same dosage will be maintained. The patients will be monitored throughout the duration of treatment by means of outpatient visits. The outcome of treatment will be documented by means of extensive non-invasive and invasive diagnostics carried out during an inpatient stay at the start of treatment and again after 24 weeks. A follow-up examination is scheduled at 8 weeks after the end of the treatment plan.

The table headed “Treatment plan” shows the different types of consultation between doctor and patient (outpatient or inpatient), the times at which they take place, and what examinations are performed.

Prior to the beginning of the study, each patient’s eligibility is verified during an outpatient visit. The inclusion criteria are thoroughly checked on this occasion, and the patient’s informed consent is obtained in writing (by signature after reading the patient information).

After a patient has been accepted for inclusion in the study, their personal data are encrypted and each patient is allocated a pseudonymised number (to serve as the patient’s “name” for screening and treatment). Only this code is used to identify the individual patient throughout the duration of treatment.

Patients whose quality of life is compromised by the disease can enrol in the study. The nationwide project will be conducted in German hospital units co-operating with the competence network. The actual treatment will take place in the same clinic(s) that the patient normally visits for treatment.

The patient information describes the various stages of the study in detail. If at the end of the project, which includes all NYHA classes, it would appear medically advisable to continue treatment with bosentan (Tracleer®) (or if the outpatient visits indicate a significant improvement in the patient’s clinical condition), it is left to the discretion of the patient’s GP to continue with regular prescriptions of Tracleer®. Alternatively, the principal investigator may recommend this course of action. Steps should then be taken at the earliest possible or most appropriate occasion to co-ordinate or organise this prescribed medical treatment (ideally by the patient’s GP or via referral to a specialist) in such a way that the bosentan therapy can be continued without interruption after the end of the studied course of treatment.

The recruitment of patients for the study closed on 31 March 2007.

A number of clinics and heart centres all over Germany are taking part in the study. An up-to-date list of these participants can be found here …