A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension

NCT ID: NCT04175600

Last Updated: 2025-11-10

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 138 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-16
• Study Completion Date: 2027-10-01

Brief Summary

The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.

Detailed Description

Pediatric PAH is a rare and progressive disorder associated with considerable morbidity and mortality. Given the significant medical need to develop treatments in children with PAH, further clinical studies in the pediatric population are therefore needed to provide more data for the management of PAH in children. Selexipag (JNJ-67896049) is an orally available, selective, and long-acting non-prostanoid agonist of the prostacyclin receptor approved and commercially available for the treatment of adult participants with PAH. Selexipag and its metabolite possess anti-fibrotic, anti-proliferative, and anti-thrombotic properties. Currently, no medicines targeting prostacyclin pathway are approved for pediatric use in PAH. An effective and orally available therapy acting on the prostacyclin receptor such as selexipag introduced at medically appropriate stage of PAH disease, and primarily in combination with current first-line oral PAH-specific medicines in participants in need of additional therapy because of insufficient disease control would represents a major advance to the therapeutic management of PAH pediatric participants. This study consists of a screening period of up to 6 weeks and a double-blind treatment period, including up-titration and maintenance periods, followed by a 3-year open-label extension period (OLEP) and a 30-day safety follow-up period that occurs after the last dose of study intervention (either double-blind or open-label). Safety, pharmacokinetic and efficacy assessments will be performed during the study. An Independent Data Monitoring Committee (IDMC) will be established to monitor data on an ongoing basis, to review interim data, and to ensure the continuing safety of the participants enrolled in this study. The approximate duration of the study is 8 years.

Conditions

Hypertension, Pulmonary

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Selexipag

Participants will receive selexipag based on the body weight on Day 1 and will continue thereafter with twice daily dosing. Selexipag will be uptitrated during the first 12 weeks until the participants reaches the individual maximum tolerated dose (iMTD) or until a maximum dose corresponding to their baseline body-weight category is achieved. Uptitration is followed by a maintenance period after Week 12 until end of treatment (EOT), at the maximum tolerated dose.

Group Type: EXPERIMENTAL

Selexipag

Intervention Type: DRUG

Selexipag tablet will be administered orally.

Placebo

Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Matching placebo tablets will be administered orally.

Interventions

Selexipag

Selexipag tablet will be administered orally.

Intervention Type: DRUG

Placebo

Matching placebo tablets will be administered orally.

Intervention Type: DRUG

Other Intervention Names

JNJ-67896049

Eligibility Criteria

Inclusion Criteria

• Participants between greater than or equal to (>=) 2 and less than (<) 18 years of age weighing >=9 kilogram (kg) at randomization
• Pulmonary arterial hypertension (PAH) diagnosis confirmed by documented historical right heart catheterization (RHC) performed at any time before participant's screening
• PAH (World Health Organization [WHO] Group 1), including participants with Down syndrome, of the following etiologies: Idiopathic PAH (IPAH); Heritable PAH (HPAH); PAH associated with congenital heart disease (PAH-associated with congenital heart disease [aCHD]) (PAH with coincidental CHD [that is, a small atrial septal defect, ventricular septal defect, or patent ductus arteriosus that does not itself account for the development of elevated PVR] and if approved by the BCAC) and Post-operative PAH (persisting / recurring/ developing >=6 months after repair of CHD); Drug or toxin-induced; PAH associated with Human immunodeficiency virus (HIV)
• WHO functional class (FC) II and III
• Participants treated with at least 1 PAH-specific treatment, example, an Endothelin receptor antagonist (ERA) and/or a Phosphodiesterase type-5 (PDE-5) inhibitor/soluble guanylate cyclase stimulator, provided that the treatment dose(s) has been stable for at least 3 months prior to first dose of study intervention

Exclusion Criteria

• PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease, and/or pulmonary capillary hemangiomatosis
• PAH associated with Eisenmenger syndrome
• Previous exposure to Uptravi (selexipag)
• Known concomitant life-threatening disease with a life expectancy <12 months
• Pregnant, planning to become pregnant, or lactating
• Known allergies, hypersensitivity, or intolerance to selexipag or its excipients

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Actelion

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Actelion Clinical Trial

Role: STUDY_DIRECTOR

Actelion

Locations

Phoenix Children's Hospital

Phoenix, Arizona, United States

UCLA Medical Center

Los Angeles, California, United States

UCSF

San Francisco, California, United States

Childrens Hospital Colorado

Aurora, Colorado, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Congenital Heart Center of the University of Florida

Gainesville, Florida, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

Detroit Medical Center

Detroit, Michigan, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Childrens Hospital Of Philadelphia

Philadelphia, Pennsylvania, United States

Texas Children's Hospital

Houston, Texas, United States

Primary Children's Hospital

Salt Lake City, Utah, United States

University of Virginia Division of Pediatric Cardiology

Charlottesville, Virginia, United States

Queensland CHILDREN'S HOSPITAL

South Brisbane, , Australia

State Institution Republican Scientific And Practical Center For Pediatric Surgery

Minsk, , Belarus

Health Institution 4Th City Children'S Clinical Hospital

Minsk, , Belarus

ULB Hôpital Erasme

Brussels, , Belgium

Universitair Ziekenhuis Gent

Ghent, , Belgium

Universitaire Ziekenhuizen Leuven

Leuven, , Belgium

Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao

Blumenau, , Brazil

Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF

Brasília, , Brazil

Hospital Pequeno Principe

Curitiba, , Brazil

Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes

Fortaleza, , Brazil

Irmandade Santa Casa de Misericordia de Porto Alegre

Porto Alegre, , Brazil

Fundacao Universitaria de Cardiologia

Porto Alegre, , Brazil

Irmandade Santa Casa de Misericordia de Sao Paulo

São Paulo, , Brazil

SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo

São Paulo, , Brazil

Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead

Sofia, , Bulgaria

Stollery Children's Hospital

Edmonton, Alberta, Canada

Hospital For Sick Children

Toronto, Ontario, Canada

Beijing Anzhen Hospital

Beijing, , China

Guangzhou Women And Children's Medical Center

Guangzhou, , China

Qingdao Women and Children's Hospital

Qingdao, , China

Shanghai Childrens Medical Center

Shanghai, , China

Children's Hospital of Fudan University

Shanghai, , China

The General Hospital of Northern Theater Command

Shenyang, , China

Clinica San Rafael

Bogotá, , Colombia

Fundacion Neumologica Colombiana

Bogotá, , Colombia

Fundacion Santa Fe de Bogota

Bogotá, , Colombia

Clínica Imbanaco S.A.S.

Cali, , Colombia

Fundacion Cardiovascular de Colombia

Piedecuesta, , Colombia

Hospital Universidad del Norte

Soledad, , Colombia

New Children's Hospital of the Helsinki University Hospital (HUS)

Helsinki, , Finland

Hôpital Cardiologique - Chru Lille

Lille, , France

Hopital de la Timone

Marseille, , France

CHU Arnaud de Villeneuve

Montpellier, , France

Hôpital Necker - Enfants Malades

Paris, , France

Hôpital Cardiologique Du Haut-Lévêque

Pessac, , France

Chu Hopital Des Enfants

Toulouse, , France

Universitätsklinikum Freiburg Zentrum

Freiburg im Breisgau, , Germany

Universitaetsklinikum Heidelberg

Heidelberg, , Germany

Herzzentrum Leipzig GmbH

Leipzig, , Germany

Klinikum der Universitaet Muenchen

München, , Germany

Gottsegen György Országos Kardiológiai Intézet

Budapest, , Hungary

Our Lady's Children's Hospital

Dublin, , Ireland

Rambam Medical Center

Haifa, , Israel

Sheba Medical Center

Ramat Gan, , Israel

Azienda Ospedaliera Policlinico S. Orsola-Malpighi

Bologna, , Italy

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Universta Degli Studi Di Padova

Padua, , Italy

Ospedale Pediatrico Bambin Gesù

Roma, , Italy

IRCCS Policlinico San Donato

S. Donato Milanese, , Italy

AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita

Torino, , Italy

Vilnius University Hospital Santariskiu Clinics

Vilnius, , Lithuania

National Heart Institute

Kuala Lumpur, , Malaysia

CICUM San Miguel

Guadalajara, , Mexico

Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas

México, , Mexico

Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)

Monterrey, , Mexico

Uniwersyteckie Centrum Kliniczne

Gdansk, , Poland

Uniwersytecki Szpital Dzieciecy w Krakowie

Krakow, , Poland

Szpital Kliniczny im Karola Jonschera

Poznan, , Poland

Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, , Poland

Wojewodzki Szpital Specjalistyczny we Wroclawiu

Wroclaw, , Poland

Slaskie Centrum Chorob Serca

Zabrze, , Poland

Uls Sao Jose - Hosp. Santa Marta

Lisbon, , Portugal

Uls Sao Joao - Hosp. Sao Joao

Porto, , Portugal

Kazan State Medical University

Kazan', , Russia

Kazan State Medical University

Kazan', , Russia

Cardiovascular Pathology Research Institute of Siberian Branch of RAMS

Kemerovo, , Russia

Childrens City Clinical Hospital n.a. Bashlyaeva

Moscow, , Russia

Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU

Moscow, , Russia

Samara Regional Clinical Cardiological Dispensary

Samara, , Russia

Univerzitetska Dečja Klinika

Belgrade, , Serbia

Seoul National University Hospital

Seoul, , South Korea

Samsung Medical Center

Seoul, , South Korea

Severance Hospital Yonsei University Health System

Seoul, , South Korea

Pusan National University Yangsan Hospital

Yangsan, , South Korea

Hosp Univ A Coruna

A Coruña, , Spain

Hosp Univ Vall D Hebron

Barcelona, , Spain

Hosp. Sant Joan de Deu

Esplugues de Llobregat, , Spain

Hosp. Gral. Univ. Gregorio Maranon

Madrid, , Spain

Hosp. Univ. La Paz

Madrid, , Spain

Hosp. Virgen Del Rocio

Seville, , Spain

Drottning Silvias barn- och ungdomssjukhus

Gothenburg, , Sweden

Skanes universitetssjukhus

Lund, , Sweden

Centre Hospitalier Universitaire Vaudois CHUV

Lausanne, , Switzerland

Kaohsiung Veterans General Hospital

Kaohsiung City, , Taiwan

National Cheng Kung University Hospital

Tainan, , Taiwan

National Taiwan University Hospital

Taipei, , Taiwan

Chiang Mai University Hospital

Chiang Mai, , Thailand

Songklanagarind hospital

Songkhla, , Thailand

Cukurova Balcali Hospital Application and Research Center

Adana, , Turkey (Türkiye)

Hacettepe University Medical Faculty

Ankara, , Turkey (Türkiye)

CAPA Istanbul University Medical Faculty

Istanbul, , Turkey (Türkiye)

Mehmet Akif Ersoy Training and Research Hospital

Istanbul, , Turkey (Türkiye)

Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital

Izmir, , Turkey (Türkiye)

Izmir Tepecik Training and Research Hospital

Izmir, , Turkey (Türkiye)

Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev

Dnipro, , Ukraine

MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'

Dnipro, , Ukraine

Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH

Kyiv, , Ukraine

MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council

Zaporizhzhya, , Ukraine

Hanoi Medical University Hospital

Hanoi, , Vietnam

Tam Anh Hospital

Ho Chi Minh City, , Vietnam

University Medical Center Ho Chi Minh city

Ho Chi Minh City, , Vietnam

Children's Hospital 1

Ho Chi Minh City, , Vietnam

Countries

Argentina; Austria; Croatia; Denmark; Romania; United States; Australia; Belarus; Belgium; Brazil; Bulgaria; Canada; China; Colombia; Finland; France; Germany; Hungary; Ireland; Israel; Italy; Lithuania; Malaysia; Mexico; Poland; Portugal; Russia; Serbia; South Korea; Spain; Sweden; Switzerland; Taiwan; Thailand; Turkey (Türkiye); Ukraine; Vietnam

Other Identifiers

AC-065A310

Identifier Type: OTHER

Identifier Source: secondary_id

2019-002817-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2022-501012-34-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

CR108716

Identifier Type: -

Identifier Source: org_study_id