A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension (SALTO)
Hypertension, Pulmonary
About this trial
This is an interventional treatment trial for Hypertension, Pulmonary
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
Sites / Locations
- Phoenix Children's Hospital
- UCLA Medical Center
- UCSF
- Childrens Hospital Colorado
- Children's National Medical Center
- Congenital Heart Center of the University of Florida
- Riley Hospital for Children
- Detroit Medical Center
- Cincinnati Children's Hospital Medical Center
- Childrens Hospital Of Philadelphia
- Texas Children's Hospital
- Primary Children's Hospital
- University of Virginia Division of Pediatric Cardiology
- Queensland CHILDREN'S HOSPITAL
- State Institution Republican Scientific And Practical Center For Pediatric Surgery
- Health Institution 4Th City Children'S Clinical Hospital
- ULB Hôpital Erasme
- Universitair Ziekenhuis Gent
- Universitaire Ziekenhuizen Leuven
- Complexo de Prevencao,Diagnostico,Terapia e Reabilitacao Respiratoria LTDA Hospital Dia do Pulmao
- Fundacao Universitaria de Cardiologia - Instituto de Cardiologia e Transplantes do DF
- Hospital Pequeno Principe
- Secretaria da Saude do Estado do Ceara - Hospital Doutor Carlos Alberto Studart Gomes
- Irmandade Santa Casa de Misericordia de Porto Alegre
- Fundacao Universitaria de Cardiologia
- Irmandade Santa Casa de Misericordia de Sao Paulo
- SPDM - Associacao Paulista para o Desenvolvimento da Medicina - Hospital Sao Paulo
- Multiprofile Hospital For Active Treatment National Cardiology Hospital, Ead
- Stollery Children's Hospital
- Hospital For Sick Children
- Beijing Anzhen Hospital
- Guangzhou Women And Children's Medical Center
- Qingdao Women and Children's Hospital
- Shanghai Children's Medical Center
- Children's Hospital of Fudan University
- The General Hospital of Northern Theater Command
- Clinica San Rafael
- Fundacion Neumologica Colombiana
- Fundacion Santa Fe de Bogota
- Clínica Imbanaco S.A.S.
- Fundacion Cardiovascular de Colombia
- Hospital Universidad del Norte
- New Children's Hospital of the Helsinki University Hospital (HUS)
- Hôpital Cardiologique - Chru Lille
- Hopital de la Timone
- CHU Arnaud de Villeneuve
- Hôpital Necker - Enfants Malades
- Hôpital Cardiologique Du Haut-Lévêque
- Chu Hopital Des Enfants
- Universitätsklinikum Freiburg Zentrum
- Universitaetsklinikum Heidelberg
- Herzzentrum Leipzig GmbH
- Klinikum der Universitaet Muenchen
- Gottsegen György Országos Kardiológiai Intézet
- Our Lady's Children's Hospital
- Rambam Medical Center
- Sheba Medical Center
- Azienda Ospedaliera Policlinico S. Orsola-Malpighi
- ASST Grande Ospedale Metropolitano Niguarda
- Universtà Degli Studi Di Padova
- Ospedale Pediatrico Bambin Gesù
- IRCCS Policlinico San Donato
- AOU Città della Salute e della Scienza di Torino, Presidio Ospedale Infantile Regina Margherita
- Pusan National University Yangsan Hospital
- Seoul National University Hospital
- Samsung Medical Center
- Severance Hospital, Yonsei University Health System
- Vilnius University Hospital Santariskiu Clinics
- National Heart Institute
- CICUM San Miguel
- Operadora de Hospitales Angeles SA de CV Hospital Angeles Lomas
- Unidad de Investigacion Clinica en Medicina S.C. (UDICEM)
- Uniwersyteckie Centrum Kliniczne
- Uniwersytecki Szpital Dzieciecy w Krakowie
- Szpital Kliniczny im. Karola Jonschera
- Instytut Pomnik - Centrum Zdrowia Dziecka
- Wojewodzki Szpital Specjalistyczny we Wroclawiu
- Slaskie Centrum Chorob Serca
- Hospital De Santa Marta
- Centro Hospitalar de São João, EPE
- Kazan State Medical University
- Kazan State Medical University
- Cardiovascular Pathology Research Institute of Siberian Branch of RAMS
- Childrens City Clinical Hospital n.a. Bashlyaeva
- Veltischev Research and Clinical Institute for Pediatrics of the Pirogov RNRMU
- Samara Regional Clinical Cardiological Dispensary
- Univerzitetska Dečja Klinika
- Hosp. Univ. A Coruña
- Hosp. Univ. Vall D Hebron
- Hosp. Sant Joan de Deu
- Hosp. Gral. Univ. Gregorio Maranon
- Hosp. Univ. La Paz
- Hosp. Virgen Del Rocio
- Drottning Silvias barn- och ungdomssjukhus
- Skanes universitetssjukhus
- Centre Hospitalier Universitaire Vaudois (CHUV)
- Kaohsiung Veterans General Hospital
- National Cheng Kung University Hospital
- National Taiwan University Hospital
- Chiang Mai University Hospital
- Songklanagarind hospital
- Cukurova Balcali Hospital Application and Research Center
- Hacettepe University Medical Faculty
- CAPA Istanbul University Medical Faculty
- Mehmet Akif Ersoy Training and Research Hospital
- Behcet Uz Pediatric Diseases and Surgery Training and Research Hospital
- Izmir Tepecik Training and Research Hospital
- Dnipropetrovsk clinical medical center of Mother and Child after prof. Rudnev
- MI 'Dnipropetrovsk Regional Clinical Center of Cardiology and Cardiac Surgery'
- Scientific Practical Medical Center for Pediatric Cardiology and Cardio Surgery of the MOH
- MI Zaporizhzhia Regional Clinical Childrens Hospital of Zaporizhzhia Regional Council
- Hanoi Medical University Hospital
- Tam Anh Hospital
- University Medical Center Ho Chi Minh city
- Children's Hospital 1
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Selexipag
Placebo
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.
Participants will receive matching placebo based on the body weight on Day 1 and will continue thereafter with twice daily dosing.