This trial is active, not recruiting.

Condition pulmonary arterial hypertension
Treatment selexipag
Phase phase 3
Sponsor Actelion
Start date July 2015
End date November 2016
Trial size 30 participants
Trial identifier NCT02471183, AC-065A304


This study enrolls patients with pulmonary arterial hypertension (PAH) treated with inhaled treprostinil. During the study, the treatment with inhaled treprostinil will be tapered off and simultaneously replaced with an oral treatment (selexipag) targeting the disease in a similar way. The purpose of the study is i) to investigate the safety and tolerability of oral selexipag in patients who transition from inhaled treprostinil, ii) to investigate the effects of oral selexipag on PAH severity and exercise ability before and after transition, and iii) to gain new information about the patients experience taking oral selexipag compared to inhaled treprostinil. Study participants may stay in the study until the FDA has granted marketing authorization.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Endpoint classification safety study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Open label
Tablet for oral administration twice a day. The individual dose will be established during the first 12 weeks of the study. Doses are in the range from 200 micrograms (1 tablet) to 1,600 micrograms (8 tablets).

Primary Outcomes

Sustained treatment transition
time frame: Baseline to Week 16
Treatment-emergent adverse events (AEs)
time frame: Baseline to EOT + 30 days
Treatment-emergent serious adverse events (SAEs)
time frame: Baseline to EOT + 30 days
AEs leading to premature discontinuation of study treatment
time frame: Baseline to EOT + 30 days
Vital signs
time frame: Baseline to EOT
Selexipag dose
time frame: Baseline to EOT
Time to discontinuation of inhaled treprostinil.
time frame: Baseline to Week 16

Secondary Outcomes

WHO functional class (FC)
time frame: Baseline to EOT
Change in 6-minute walk distance (6MWD)
time frame: Baseline to Week 16
Proportion of patients with change in 6MWD
time frame: Baseline to Week 16
N-terminal pro b-type natriuretic peptide (NT-proBNP)
time frame: Baseline to Week 16

Eligibility Criteria

Male or female participants from 18 years up to 75 years old.

Inclusion Criteria: - Male and female patients aged from 18 to 75 years (inclusive) with PAH. - Etiology of PAH belonging to one of the following subgroups of Group 1 PAH according to Nice classification: idiopathic PAH, Heritable PAH, drug or toxin induced, associated with connective tissue disease, associated with HIV infection, associated with congenital heart disease with simple systemic-to-pulmonary shunt at least 1 year after surgical repair. - Women of childbearing potential are eligible only if the following apply: Negative serum pregnancy test at Visit 1 and a negative urine pregnancy test at Visit on Day 1, agreement to undertake monthly urine pregnancy tests during the study and up to 30 days after study drug discontinuation, agreement to use efficient methods of birth control from Visit 1 up to at least 30 days after study treatment discontinuation. - Documented hemodynamic diagnosis of PAH by right heart catheterization (RHC). - Inhaled treprostinil treatment ongoing for at least 90 days and at stable dose for at least 30 days prior to Day 1. - WHO functional class (FC) II or III at Visit 1 and Visit 2. - 6-minute walk distance (6MWD) ≥ 300 m at Visit 1. - On background oral PAH therapy for at least 90 days and on a stable dose for 30 days prior to Visit 2. Acceptable concomitant PAH therapies are one or two of the following: a) Endothelin receptor antagonist (ERA), b) Phosphodiesterase type 5 (PDE-5) inhibitor or soluble guanylate cyclase (sGC) stimulator. Exclusion Criteria: - Treatment with any prostacyclin or prostacyclin analogs other than inhaled treprostinil within 90 days before Day 1, or patients scheduled to receive any of these treatments within the duration of the study. - Any hospitalization within 90 days before Day 1. - Worsening in WHO FC within 30 days prior to Day 1. - At any time prior to Day 1, documented moderate or severe obstructive or restrictive lung disease. - Known or suspicion of pulmonary veno-occlusive disease (PVOD). - Anemia: < 80 g/L (5.0 mmol/L) hemoglobin. - Clinically relevant thyroid disease (hypo- or hyperthyroidism). - Known and documented severe hepatic impairment. - Uncontrolled hypertension. - Sitting systolic BP < 85 mmHg. - Acute myocardial infarction within the last 90 days prior to Visit 1. - History of left-sided heart disease. - Left ventricular disease/dysfunction risk factors. - Documented pericardial effusion within 90 days prior to Visit 1. - Documented severe renal insufficiency. - Receiving or having received any investigational drugs within 90 days before Day 1. - Having received selexipag at any time before Day 1. - Acute or chronic impairment (other than dyspnea), limiting the ability to comply with study requirements. - Recently conducted or planned cardio-pulmonary rehabilitation program based on exercise training during the study. - Psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements. - Known concomitant life-threatening disease with a life expectancy < 12 months. - Females who are lactating or pregnant or plan to become pregnant during the study. - Known hypersensitivity to any of the excipients of the drug formulation.

Additional Information

Official title Multicenter, Open-label, Single-group Study to Assess the Tolerability and the Safety of the Transition From Inhaled Treprostinil to Oral Selexipag in Adult Patients With Pulmonary Arterial Hypertension
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Actelion.