A study of the efficiacy and safety of selexipag in patients with SAPH
A multicentre, randomised, double-blind, placebo-controlled study in participants with sarcoidosis-associated pulmonary hypertension (SAPH) to assess the efficacy and safety of oral selexipag.
Actelion Pharmaceuticals Ltd
Duration of Study in the UK
5 years, 5 months, 6 days
Sarcoidosis is a disease involving abnormal collections of inflammatory cells that form lumps known as granulomas, affecting particularly the lungs and lymphatic system. Pulmonary hypertension (PH), is a rare disorder that causes increases in blood pressure (pulmonary vascular pressure - PVR) within the arteries of the lungs. PH is increasingly recognised as a serious complication of pulmonary sarcoidosis and is associated with an increased morbidity and mortality.
Severe untreated PH carries a poor prognosis and is associated with higher mortality in patients with interstitial lung diseases and sarcoidosis. Currently there are no approved therapies available to treat SAPH (sarcoidosis-associated pulmonary hypertension) patients; PH specific therapies are used. Small studies using PH specific therapies have shown that PVR is reduced in SAPH patients.
This will be the first global, randomised, controlled study to explore if Selexipag is effective and safe for treating patients with sarcoidosis-associated pulmonary hypertension (SAPH).
The primary endpoint of the study is to demonstrate that Selexipag is more effective in reducing pulmonary vascular hypertension after 20 weeks of treatment, compared to 20 weeks of treatment with placebo.
The study will be conducted in four phases, consisting of a screening period of 8-30 days, a double-blind treatment period of 12 months, an open-label extension period of 24 months and a safety follow up period of 30 days. The duration of the study is expected to be up to 3 years and 2 months.
Patients diagnosed with SAPH and deemed eligible to participate will be enrolled into two sequential cohorts. The first 86 participants randomised will become the hemodynamic cohort, and the remaining participants the non-hemodynamic cohort.
The study will be conducted at NHS specialist PH centres in the United Kingdom.
East Midlands - Leicester South Research Ethics Committee
Date of REC Opinion
14 Aug 2019
Further Information Favourable Opinion