A Study in Participants With Sarcoidosis-associated Pulmonary Hypertension (SAPH) to Assess the Efficacy and Safety of Oral Selexipag



Sponsor:   Actelion


Sommario: 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).




TIPOLOGIA STUDIO

 
Interventistico



FASE

 
Fase 2




TRATTAMENTO

  
    
  
  • Selexipag
  • Placebo
    • 






OBIETTIVO PRIMARIO


  
  • Misura: Pulmonary Vascular Resistance (PVR) up to Week 26
  • Tempo: Baseline up to Week 26
  • Descrizione: PVR represents the resistance against which the right ventricle needs to pump. PVR was
determined by right heart catheterization (RHC). It was measured as the ratio of the PVR
value post-treatment initiation up to Week 26 (post) versus the PVR value pre-treatment
initiation at baseline (pre), expressed as a percentage of baseline value. The baseline
reference value for PVR was based on the last RHC performed prior to study intervention
initiation. PVR was calculated as 80*(mean pulmonary arterial pressure - pulmonary artery
wedge pressure) divided by cardiac output. As specified in the statistical analysis plan,
data was not planned to be summarized for this outcome measure and only individual
participant wise data was collected.






CRITERI DI ELIGIBILITA'


    
             
  • 
        Criteri di inclusione e esclusione
        
    Main Inclusion Criteria:

  -  Confirmed diagnosis of sarcoidosis as per American Thoracic Society (ATS) criteria

  -  Sarcoidosis-associated precapillary PH, confirmed by RHC (at rest) within 90 days
     prior to randomization.

  -  PH severity according to modified WHO FC II-IV at Screening and randomization;
     participants in WHO FC IV must be in a stable condition and able to perform a 6MWT.

  -  Either not receiving treatment with PH-specific treatment or oral PH-specific
     monotherapy (ie, riociguat or PDE5i or ERA); if on oral PH-specific monotherapy then
     treatment had to be stable (ie, no introduction of new therapies or changes in dose)
     for at least 90 days prior to both and the RHC qualifying for enrollment and
     randomization

  -  Stable sarcoidosis treatment regimen, ie, no new specific anti-inflammatory
     treatment for sarcoidosis for at least 90 days, and stable dose(s) for at least 30
     days prior to both the RHC qualifying for enrollment and randomization

  -  6-minute walk distance (6MWD) greater than or equal to (>=) 50 meters both at
     Screening and at the time of randomization. Participants can use their usual walking
     aids during the test (example, cane, crutches). The same walking aid should be used
     for all 6-minute walk test (6MWTs). Walkers are not allowed

  -  Forced Vital Capacity (FVC) greater than (>) 50 percent (%) and Forced Expiratory
     Volume (in 1 second) (FEV1) > 50% of predicted at Screening

  -  Diffusing capacity of the lung for carbon monoxide (DLCO) >= 40% of predicted. If
     DLCO less than (<) 40% of predicted, the extent of emphysema should not be greater
     than that of fibrosis as assessed by high resolution computerized tomography (CT)
     scan

  -  Women of childbearing potential must have a negative pregnancy test at screening and
     randomization, must agree to undertake monthly urine pregnancy tests, and to
     practice an acceptable method of contraception and agreeing to remain on an
     acceptable method while receiving study intervention and until 30 days after last
     dose of study intervention

  -  A woman only using hormonal contraceptives must have been using this method for at
     least 30 days prior to randomization

Main Exclusion Criteria:

  -  PH due to left heart disease (PAWP >15 mmHg).

  -  History of left heart failure (LHF) as assessed by the investigator including
     cardiomyopathies, and cardiac sarcoidosis, with a left ventricular ejection fraction
     (LVEF) <40%.

  -  Treatment with prostacyclin, prostacyclin analogues or IP receptor agonists (ie,
     selexipag) within 90 days prior to randomization and/or prior to the RHC qualifying
     for enrollment, except those given at vasodilator testing during RHC.

  -  SBP <90 mmHg at Screening or at randomization.

  -  Included on a lung transplant list or planned to be included until Visit 6 / Week
     39.

  -  Change in dose or initiation of new diuretics and/or calcium channel blockers within
     1 week prior to RHC qualifying for enrollment.

  -  Any condition for which, in the opinion of the investigator, participation would not
     be in the best interests of the participant (eg, compromise well-being), or that
     could prevent, limit, or confound the protocol-specified assessments.

  -  Any acute or chronic impairment that may influence the ability to comply with study
     requirements such as to perform RHC, a reliable and reproducible 6MWT, or lung
     function tests.

  -  Any other criteria as per selexipag Summary of Product Characteristics (SmPC)
    
        
    • 
    




SESSO

  
Tutti




  


ETA' MINIMA



Età Minima: 18 Years

Età Massima: 75 Years






LUOGO


  
Ospedale S.Giuseppe, Gruppo MultiMedica
Milan	6951411, 20123
Fondazione Maugeri Montescano
Pavia	3171366, 27100
Umberto I Pol. di Roma-Università di Roma La Sapienza
Roma	8957247, 00165
Universita Cattolica del Sacro Cuore - Fondazione Policlinico Universitario 'A. Gemelli'
Roma	8957247, 00168
A.O.U. Città della Salute e della Scienza
Torino	8980539, 10126