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

Study Purpose

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).

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years - 75 Years
Gender All
More Inclusion & Exclusion Criteria

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)

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03942211
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Actelion
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Rainer Zimmermann
Principal Investigator Affiliation Actelion
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Belgium, Brazil, Canada, France, Germany, Italy, Netherlands, Spain, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Sarcoidosis-associated Pulmonary Hypertension
Additional Details

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.

Arms & Interventions

Arms

Experimental: Selexipag 200 micro gram (μg)

Study intervention will be up-titrated to allow each participant to reach their individual maximum tolerated dose (iMTD), in the range of 200 μg to1600 μg (ie, 1 to 8 tablets) twice daily/once daily. Dosing frequency will be twice daily, except for participants with moderate hepatic impairment (Child-Pugh Class B) or who are concomitantly taking (a) moderate CYP2C8 inhibitor(s), who receive study intervention once daily. The dose will be up-titrated by the investigator/delegate in 200 μg twice daily/once daily increments at weekly intervals during scheduled TCs until reaching the iMTD. If the dose regimen is not well tolerated or symptoms cannot be fully managed with symptomatic treatment, the duration of the titration step can be prolonged to 2 weeks. If needed, the dose can be reduced by 200 μg twice daily/once daily.

Placebo Comparator: Placebo

The comparator will be administered similarly to the experimental intervention.

Interventions

Drug: - Selexipag

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

Drug: - Placebo

Oral tablets without active compound. Participants can receive 1 to 8 tablets at each administration.

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Cedars Sinai Heart Institute, Beverly Hills, California

Status

Withdrawn

Address

Cedars Sinai Heart Institute

Beverly Hills, California, 90211

David Geffen School of Medicine at UCLA, Los Angeles, California

Status

Withdrawn

Address

David Geffen School of Medicine at UCLA

Los Angeles, California, 90024

Keck School of Medicine of USC, Los Angeles, California

Status

Not yet recruiting

Address

Keck School of Medicine of USC

Los Angeles, California, 90033

UC Davis Medical Center, Sacramento, California

Status

Withdrawn

Address

UC Davis Medical Center

Sacramento, California, 95817-2201

National Jewish Health, Denver, Colorado

Status

Withdrawn

Address

National Jewish Health

Denver, Colorado, 80206

MedStar Georgetown University Hospital, Washington, District of Columbia

Status

Withdrawn

Address

MedStar Georgetown University Hospital

Washington, District of Columbia, 20007

The GW Medical Faculty Associates, Washington, District of Columbia

Status

Withdrawn

Address

The GW Medical Faculty Associates

Washington, District of Columbia, 20037

University of Florida, Gainesville, Florida

Status

Not yet recruiting

Address

University of Florida

Gainesville, Florida, 32610

Jacksonville, Florida

Status

Withdrawn

Address

St Vincents Lung, Sleep And Critical Care

Jacksonville, Florida, 32004

Mayo Clinic, Jacksonville, Florida

Status

Withdrawn

Address

Mayo Clinic

Jacksonville, Florida, 32224

University of Miami, Miami, Florida

Status

Withdrawn

Address

University of Miami

Miami, Florida, 33125

Emory University, Atlanta, Georgia

Status

Withdrawn

Address

Emory University

Atlanta, Georgia, 30322

Chicago, Illinois

Status

Withdrawn

Address

University Of Il Sarcoidosis Translational Advanced Research Center Star

Chicago, Illinois, 60612

University of Chicago, Chicago, Illinois

Status

Not yet recruiting

Address

University of Chicago

Chicago, Illinois, 60637

St. Vincent Medical Group, Inc., Indianapolis, Indiana

Status

Recruiting

Address

St. Vincent Medical Group, Inc.

Indianapolis, Indiana, 46260

LSU Health Sciences Center New Orleans, New Orleans, Louisiana

Status

Recruiting

Address

LSU Health Sciences Center New Orleans

New Orleans, Louisiana, 70112

Shreveport, Louisiana

Status

Withdrawn

Address

Louisiana State University Health Sciences Center

Shreveport, Louisiana, 71103

Detroit, Michigan

Status

Withdrawn

Address

Wayne State University/Detroit Medical Center

Detroit, Michigan, 48201-2153

Albany, New York

Status

Withdrawn

Address

Albany Medical Center (AMC) - Pulmonary and Critical Care Medicine

Albany, New York, 12208

NYU Langone Health, New York, New York

Status

Withdrawn

Address

NYU Langone Health

New York, New York, 10016

Icahn School of Medicine at Mount Sinai, New York, New York

Status

Recruiting

Address

Icahn School of Medicine at Mount Sinai

New York, New York, 10029

Chapel Hill, North Carolina

Status

Recruiting

Address

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514

Duke University Medical Center, Durham, North Carolina

Status

Recruiting

Address

Duke University Medical Center

Durham, North Carolina, 27710

University of Cincinnati, Cincinnati, Ohio

Status

Completed

Address

University of Cincinnati

Cincinnati, Ohio, 45267

Cleveland Clinic, Cleveland, Ohio

Status

Recruiting

Address

Cleveland Clinic

Cleveland, Ohio, 44195-0001

Columbus, Ohio

Status

Withdrawn

Address

Dorothy M. Davis Heart & Lung Research Institute

Columbus, Ohio, 43210

Philadelphia, Pennsylvania

Status

Withdrawn

Address

Lewis Katz School of Medicine, Temple University

Philadelphia, Pennsylvania, 19140

Charleston, South Carolina

Status

Recruiting

Address

Medical University of South Carolina (MUSC) - College of Medicine (COM)

Charleston, South Carolina, 29425-8900

University of Tennessee, Memphis, Tennessee

Status

Withdrawn

Address

University of Tennessee

Memphis, Tennessee, 37920

Dallas, Texas

Status

Withdrawn

Address

University of Texas Southwestern Medical Center

Dallas, Texas, 75390-9016

Houston Methodist Hospital, Houston, Texas

Status

Withdrawn

Address

Houston Methodist Hospital

Houston, Texas, 77030

University of Texas Houston, Houston, Texas

Status

Withdrawn

Address

University of Texas Houston

Houston, Texas, 77030

INOVA, Falls Church, Virginia

Status

Withdrawn

Address

INOVA

Falls Church, Virginia, 22042

International Sites

Universitaire Ziekenhuizen Leuven, Leuven, Belgium

Status

Recruiting

Address

Universitaire Ziekenhuizen Leuven

Leuven, , 3000

Fortaleza, Brazil

Status

Recruiting

Address

Hospital de Messejana dr. Carlos Alberto Studart Gomes

Fortaleza, , 60840-285

Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil

Status

Recruiting

Address

Hospital de Clínicas de Porto Alegre

Porto Alegre, , 90.035-903

Porto Alegre, Brazil

Status

Not yet recruiting

Address

Santa Casa de Misericordia de Porto Alegre

Porto Alegre, , 90035-074

Instituto do Coracao (INCOR) - HCFMUSP, São Paulo, Brazil

Status

Recruiting

Address

Instituto do Coracao (INCOR) - HCFMUSP

São Paulo, , 05403000

London Health Sciences Centre, London, Ontario, Canada

Status

Recruiting

Address

London Health Sciences Centre

London, Ontario, N6A 5W9

Toronto, Ontario, Canada

Status

Withdrawn

Address

University Health Network - Toronto General Hospital

Toronto, Ontario, M5G 2N2

Jewish General Hospital, Montreal, Quebec, Canada

Status

Recruiting

Address

Jewish General Hospital

Montreal, Quebec, H3T 1E2

Hôpital Avicenne, Bobigny, France

Status

Recruiting

Address

Hôpital Avicenne

Bobigny, , 93000

Bron Cedex, France

Status

Recruiting

Address

GH est - Hôpital Cardiovasculaire et Pneumologie Louis Pradel

Bron Cedex, , 69677

Hôpital Kremlin Bicêtre, Le Kremlin Bicetre Cedex, France

Status

Recruiting

Address

Hôpital Kremlin Bicêtre

Le Kremlin Bicetre Cedex, , 94270

CHR Hôpital Calmette, Lille Cedex, France

Status

Withdrawn

Address

CHR Hôpital Calmette

Lille Cedex, , 59037

Hopital Nord, Marseille cedex 20, France

Status

Recruiting

Address

Hopital Nord

Marseille cedex 20, , 13915

CHU de Nancy - Hôpital de Brabois, Vandoeuvre les Nancy Cedex, France

Status

Recruiting

Address

CHU de Nancy - Hôpital de Brabois

Vandoeuvre les Nancy Cedex, , 54511

Evangelische Lungenklinik Berlin, Berlin, Germany

Status

Recruiting

Address

Evangelische Lungenklinik Berlin

Berlin, , 13125

Universitatsklinikum Bonn, Bonn, Germany

Status

Completed

Address

Universitatsklinikum Bonn

Bonn, , 53105

Dresden, Germany

Status

Active, not recruiting

Address

Universitatsklinikum Carl Gustav Carcus Dresden

Dresden, , 01307

Universitätsmedizin Greifswald, Greifswald, Germany

Status

Withdrawn

Address

Universitätsmedizin Greifswald

Greifswald, , 17475

Thoraxklinik Heidelberg, Heidelberg, Germany

Status

Active, not recruiting

Address

Thoraxklinik Heidelberg

Heidelberg, , 69126

Universitaetsklinikum des Saarlandes, Homburg, Germany

Status

Withdrawn

Address

Universitaetsklinikum des Saarlandes

Homburg, , 66421

Universitätsklinikum Schleswig-Holstein, Luebeck, Germany

Status

Recruiting

Address

Universitätsklinikum Schleswig-Holstein

Luebeck, , 23538

Kardiologische Praxis Papenburg, Papenburg, Germany

Status

Withdrawn

Address

Kardiologische Praxis Papenburg

Papenburg, , 26871

Universitaetsklinikum Regensburg, Regensburg, Germany

Status

Recruiting

Address

Universitaetsklinikum Regensburg

Regensburg, , 93053

Solingen, Germany

Status

Withdrawn

Address

Wissenschaftliches Institut Bethanien für Pneumologie e.V

Solingen, , 42699

Stuttgart, Germany

Status

Recruiting

Address

RBK Lungenzentrum Stuttgart am Robert-Bosch-Krankenhaus

Stuttgart, , 70839

Würzburg, Germany

Status

Active, not recruiting

Address

Klinikum Würzburg Mitte gGmbH Standort Missioklinik

Würzburg, , 97074

AUSL della Romagna - Ospedale di Forlì, Forlì, Italy

Status

Withdrawn

Address

AUSL della Romagna - Ospedale di Forlì

Forlì, , 47121

Ospedale S.Giuseppe, Gruppo MultiMedica, Milano, Italy

Status

Recruiting

Address

Ospedale S.Giuseppe, Gruppo MultiMedica

Milano, , 20123

Palermo, Italy

Status

Withdrawn

Address

ISMETT Istituto Mediterraneo Trapianti e Terapie ad Alta Specializzazione

Palermo, , 90127

Fondazione Maugeri Montescano, Pavia, Italy

Status

Recruiting

Address

Fondazione Maugeri Montescano

Pavia, , 27100

Roma, Italy

Status

Recruiting

Address

Umberto I Pol. di Roma-Università di Roma La Sapienza

Roma, , 00165

Roma, Italy

Status

Recruiting

Address

Universita Cattolica del Sacro Cuore - Fondazione Policlinico Universitario 'A. Gemelli'

Roma, , 00168

Torino, Italy

Status

Recruiting

Address

A.O.U. Città della Salute e della Scienza

Torino, , 10126

Hospital Ca' Foncello, Treviso, Italy

Status

Withdrawn

Address

Hospital Ca' Foncello

Treviso, , 31100

Trieste, Italy

Status

Withdrawn

Address

Ospedale di Cattinara - Struttura complessa di Pneumologia

Trieste, , 34149

VUMC Amsterdam, Amsterdam, Netherlands

Status

Recruiting

Address

VUMC Amsterdam

Amsterdam, , 1081 HV

Sint Antonius Ziekenhuis, Nieuwegein, Netherlands

Status

Recruiting

Address

Sint Antonius Ziekenhuis

Nieuwegein, , 3435 CM

Hosp. Clinic I Provincial de Barcelona, Barcelona, Spain

Status

Recruiting

Address

Hosp. Clinic I Provincial de Barcelona

Barcelona, , 08036

Hosp. Univ. Vall D Hebron, Barcelona, Spain

Status

Withdrawn

Address

Hosp. Univ. Vall D Hebron

Barcelona, , 8035

Hosp. Univ. Marques de Valdecilla, Santander, Spain

Status

Recruiting

Address

Hosp. Univ. Marques de Valdecilla

Santander, , 39008

Royal Free Hospital, London, United Kingdom

Status

Recruiting

Address

Royal Free Hospital

London, , NW3 2QG

Royal Brompton Hospital, London, United Kingdom

Status

Recruiting

Address

Royal Brompton Hospital

London, , SW3 6NP

Sheffield, United Kingdom

Status

Withdrawn

Address

Sheffield Teaching Hospitals NHS Foundation Trust Royal Hallamshire Hospital

Sheffield, , S10 2RX