A Study in Participants With Sarcoidosis-associated Pulmonary Hypertension (SAPH) to Assess the Efficacy and Safety of Oral Selexipag
Condition: Sarcoidosis-associated Pulmonary Hypertension
MeSH terms: Hypertension, Pulmonary; Hypertension; Sarcoidosis
Study type: Interventional
Phase: Phase 2

Summary: 
      Oral selexipag is commercially available in several countries for the treatment of a
      particular group of pulmonary hypertension (PH) called pulmonary arterial hypertension (PAH).
      The aim of the present study is to investigate whether selexipag could be helpful to treat
      patients with another form of PH called sarcoidosis-associated pulmonary hypertension (SAPH).
    

Detailed description: 
      Pulmonary hypertension (PH) is a pathophysiological disorder that may involve multiple
      clinical conditions and can complicate several cardiovascular and respiratory diseases.
      Sarcoidosis is a multisystemic disorder that is characterized by non-caseating granulomas
      which are present in multiple tissues, particularly in the lung and lymphatic system. Severe
      untreated pulmonary hypertension (PH) carries a poor prognosis and is associated with higher
      mortality in participants with interstitial lung diseases and sarcoidosis. While there is no
      approved treatment for SAPH, PH-specific treatments are frequently used. Selexipag is a
      selective, orally available and long-acting non-prostanoid agonist of the prostacyclin
      receptor (prostacyclin [IP] receptor) for the treatment of patients with PAH. The rationale
      for this study is based on the unmet medical need for new therapeutic options for patients
      with SAPH and is supported by the established efficacy and safety of selexipag in the PAH
      indication, the shared pathomechanism between SAPH and PAH, and the available data on the
      efficacy and safety of PH-specific therapies in SAPH. This study consists of screening
      period, main observation period and double blind extension period and safety follow-up
      period. The duration of individual participation in the study will be different for each
      individual participant (between approximately 15 months and up to approximately 3.5 years)
      and will depend on the time of each participant's individual date of entering the study and
      the total recruitment time. The efficacy assessments include right heart catheterization
      (RHC), assessment of exercise capacity, dyspnea, pulmonary function tests, etc. Safety and
      tolerability will be evaluated throughout the study and includes review of concomitant
      medications and adverse events (AEs), clinical laboratory tests, 12-lead electrocardiogram
      (ECG), vital signs, physical examination, and pregnancy testing.
    

Intervention name(s): Selexipag; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Oral tablets containing 200 µg of selexipag. Depending on the iMTD, participants will receive 1 (200 µg) to 8 (1600 µg) tablets at each administration; Oral tablets without active compound. Participants can receive 1 to 8 tablets at each administration.
Other intervention name(s): JNJ-678896049; ACT-293987
Intervention MeSH terms: Selexipag
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: selexipag, sarcoidosis, pulmonary hypertension