Sarcoidosis-associated Pulmonary Hypertension Clinical Trial
— SPHINXOfficial title:
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Sarcoidosis-Associated Pulmonary Hypertension (SAPH) to Assess the Efficacy and Safety of Oral Selexipag.
| Verified date | April 2024 |
| Source | Actelion |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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).
| Status | Terminated |
| Enrollment | 10 |
| Est. completion date | April 19, 2023 |
| Est. primary completion date | April 19, 2023 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 75 Years |
| Eligibility | 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) |
| Country | Name | City | State |
|---|---|---|---|
| Belgium | Universitaire Ziekenhuizen Leuven | Leuven | |
| Brazil | Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes | Fortaleza | |
| Brazil | Hospital das Clinicas de Porto Alegre | Porto Alegre | |
| Brazil | Hospital Das Clinicas Da Faculdade De Medicina Da USP | Sao Paulo | |
| Canada | London Health Sciences Centre | London | Ontario |
| Canada | Jewish General Hospital | Montreal | Quebec |
| France | Hôpital Avicenne | Bobigny | |
| France | GH est - Hôpital Cardiovasculaire et Pneumologie Louis Pradel | Bron Cedex | |
| France | Hôpital Kremlin Bicêtre | Le Kremlin Bicetre Cedex | |
| France | Hopital Nord | Marseille cedex 20 | |
| France | CHU de Nancy - Hopital de Brabois | Vandoeuvre les Nancy Cedex | |
| Germany | Evangelische Lungenklinik Berlin | Berlin | |
| Germany | Universitatsklinikum Bonn | Bonn | |
| Germany | Universitatsklinikum Carl Gustav Carcus Dresden | Dresden | |
| Germany | Thoraxklinik Heidelberg | Heidelberg | |
| Germany | Universitatsklinikum Schleswig Holstein | Luebeck | |
| Germany | Universitaetsklinikum Regensburg | Regensburg | |
| Germany | RBK Lungenzentrum Stuttgart am Robert-Bosch-Krankenhaus | Stuttgart | |
| Germany | Klinikum Würzburg Mitte gGmbH Standort Missioklinik | Würzburg | |
| Italy | Ospedale S.Giuseppe, Gruppo MultiMedica | Milano | |
| Italy | Fondazione Maugeri Montescano | Pavia | |
| Italy | Umberto I Pol. di Roma-Università di Roma La Sapienza | Roma | |
| Italy | Universita Cattolica del Sacro Cuore - Fondazione Policlinico Universitario 'A. Gemelli' | Roma | |
| Italy | A.O.U. Città della Salute e della Scienza | Torino | |
| Netherlands | VUMC Amsterdam | Amsterdam | |
| Netherlands | Sint Antonius Ziekenhuis | Nieuwegein | |
| Spain | Hosp. Clinic de Barcelona | Barcelona | |
| Spain | Hosp. Univ. Marques de Valdecilla | Santander | |
| United Kingdom | Royal Brompton Hospital | London | |
| United Kingdom | Royal Free Hospital | London | |
| United States | University of North Carolina at Chapel Hill | Chapel Hill | North Carolina |
| United States | Medical University of South Carolina (MUSC) - College of Medicine (COM) | Charleston | South Carolina |
| United States | University of Cincinnati | Cincinnati | Ohio |
| United States | Cleveland Clinic | Cleveland | Ohio |
| United States | Duke University Medical Center | Durham | North Carolina |
| United States | St. Vincent Medical Group, Inc. | Indianapolis | Indiana |
| United States | LSU Health Sciences Center New Orleans | New Orleans | Louisiana |
| United States | Icahn School of Medicine at Mount Sinai | New York | New York |
| Lead Sponsor | Collaborator |
|---|---|
| Actelion |
United States, Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Pulmonary Vascular Resistance (PVR) up to Week 26 | 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. | Baseline up to Week 26 |