None listed
Conditions
Interventions
Group 1 Type of group
ACT-064992 in tablets, dose of 3 mg, once a day.
Group name:Group 3 Type of group
Placebo similar to ACT-064992, once a day.
Sponsors
Actelion Pharmaceuticals Ltd,
Eligibility
Sex/Gender
All
Age
12 Years to 90 Years
Inclusion criteria
Inclusion criteria: • Signed informed consent before initiating any procedure specific to the study. • Patients with symptomatic pulmonary arterial hypertension (PAH) in modified functional classes II to IV of WHO. • Patients with the following types of PAH: Idiopathic (IPAH); Family (FPAH) • The diagnosis of PAH confirmed by hemodynamic evaluation performed before randomization • Walking distance of 6 minutes (6MWD)> 50 m and 12 years of age (women with pregnancy potential should have a negative pregnancy test before starting treatment and should use a reliable contraceptive method).
Exclusion criteria
Exclusion criteria: • PAH associated with portal hypertension, thyroid disorders, glycogen storage disease, Gaucher´s disease, hereditary hemorrhagic telangiectasia, hemoglobinopathies, myeloproliferative disorders and splenectomy. • PAH associated with simple congenital systemic to pulmonary uncorrected shunts, and combined and complex systemic to pulmonary shunts, corrected or uncorrected. • PAH associated with significant venous or capillary involvement (PCWP> 15 mmHg), known pulmonary veno-occlusive disease and capillary pulmonary hemangiomatosis. • Persistent pulmonary hypertension in the newborn. • Pulmonary arterial hypertension of groups 2 to 5 of the Venice classification. • Moderate to severe obstructive pulmonary disease: volume volume in first second / forced vital capacity (FEV1 / FVC) 1.5 times the upper limit of normal. • Hemoglobin <75% of the lower limit of the normal range. • Systolic blood pressure <100 mmHg. • Acute or chronic physical impairment (other than dyspnea), which limits the ability to meet the requirements of the study. • Pregnant or lactating women. • Concomitant disease that puts life expectancy at risk <12 months. • Body weight <40 kg. • Any condition that avoids adherence to the protocol or therapy. • Cardiopulmonary rehabilitation program based on the recently initiated or planned short-term exercise (<8 weeks before randomization). • Treatment with endothelin receptor antagonists (ERAs) in the 3 months prior to randomization. • Systemic treatment in the week prior to randomization with cyclosporin A or tacrolimus, everolimus, sirolimus (calcineurin inhibitors or mTOR inhibitors). • Known hypersensitivity to drugs of the same class as the study drug, or to any of its excipients. • Treatment planned, or in progress, with another investigational drug in the month prior to randomization.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Outcome name:Defined as follows: Death, Atrial septostomy, Lung transplantation, Start of intravenous or subcutaneous prostanoids (e.g., epoprostenol, treprostinil), another worsening of pulmonary arterial hypertension. Measure:Time from the start of treatment to the first event of morbidity or mortality Timepoints:After treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Outcome name:The 6MWT is evaluated in the Selection, Randomization, Month 3, Month 6, every 6 months thereafter and EOT. It is a non-encouraged test, which measures the distance traveled in a 6-minute walk. Measure:Change of the baseline value to 6 months in the 6MWD Timepoints:6 months ; Outcome name:The WHO functional class of PAH is evaluated in the Selection, Randomization, Month 3, Month 6, every 6 months thereafter and EOT Measure:Change in baseline to Month 6 in modified functional class OMS Timepoints:6 months ; Outcome name:Death due to PAH, or the appearance of an AE caused by the treatment that led to the permanent interruption of the study treatment with a fatal outcome due to PAH within 4 weeks after the interruption of the study treatment, or Hospitalization by PAH Measure:Time to death due to PAH or hospitalization for PAH to EOT Timepoints:During and after treatment ; Outcome name:Death due to PAH, or the appearance of an AE caused by the treatment that led to the permanent interruption of the study treatment with a fatal outcome due to PAH within 4 weeks after the interruption of the study treatment, or Hospitalization by PAH Measure:Time to death for all causes until the EOT or time to the appearance of an AE caused by the treatment that led to the permanent interruption of the study treatment with a fatal outcome within 4 weeks after the interruption of the study treatment . Timepoints:4 weeks after the interruption of the study treatment. | — |
Countries
Austria, Belgium, Bulgaria, Finland, France, Germany, Italy, Netherlands, Peru, Portugal, Sweden, United Kindgdom
Contacts
Public ContactFelix Medina
SYNEOS HEALTH PERU S.R.L.
Outcome results
None listed