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

NCT04175600 · tracked via the Priya Life Science Germany tracker

Sponsor

Actelion

Phase

Phase 3

Started

2020-01-16

Last updated

2026-08-28

Condition(s) studied

Hypertension, Pulmonary

Investigational drug(s) / intervention(s)

Selexipag →PlaceboStandard of Care (SOC): Endothelin receptor antagonistSOC: Phosphodiesterase type 5 (PDE-5) inhibitorSOC: Soluble guanylate cyclase stimulator

Selexipag: Selexipag tablet will be administered orally.

Placebo: Matching placebo tablets will be administered orally.

Standard of Care (SOC): Endothelin receptor antagonist: ERAs will be administered as SOC therapy.

SOC: Phosphodiesterase type 5 (PDE-5) inhibitor: PDE-5 inhibitor will be administered as SOC therapy.

SOC: Soluble guanylate cyclase stimulator: Soluble guanylate cyclase stimulator will be administered as SOC therapy.

Study 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.

Eligibility

Sex

ALL

Min age

2 Years

Max age

17 Years

Healthy volunteers

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

Primary outcome measure(s)

Double-blind Period: Time to Disease Progression — Placebo arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 199 weeks); Selexipag arm: Randomization (Day 1) up to 7 days after last dose in the DB treatment (maximum up to 213 weeks)
Time to disease progression: time from randomization to first Clinical Event Committee (CEC)-confirmed disease progression event occurring between date of randomization until double-blind end date (DBED) + 7 days. Disease progression event as adjudicated by CEC, defined as any of the following: death (all causes); atrial septostomy or Potts' anastomosis, or registration on lung transplant list; hospitalization due to worsening pulmonary arterial hypertension (PAH); clinical worsening of PAH. Clinical worsening of PAH was defined as initiation of new PAH-specific therapy or intravenous diuretics or continuous oxygen use and at least 1 of the following: worsening in World Health Organization (WHO) functional class (FC); new occurrence or worsening: of syncope, of at least 2 PAH symptoms (shortness of breath/dyspnea, chest pain, cyanosis, dizziness/near syncope, or fatigue), of signs of right heart failure not responding to oral diuretics. Kaplan-Meier method was used for estimation.

Trial sites (116)

Facility	City	Region
Phoenix Children's Hospital	Phoenix	Arizona
UCLA Medical Center	Los Angeles	California
UCSF	San Francisco	California
Childrens Hospital Colorado	Aurora	Colorado
Children's National Medical Center	Washington D.C.	District of Columbia
Congenital Heart Center of the University of Florida	Gainesville	Florida
Riley Hospital for Children	Indianapolis	Indiana
Detroit Medical Center	Detroit	Michigan
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
Childrens Hospital Of Philadelphia	Philadelphia	Pennsylvania
Texas Children's Hospital	Houston	Texas
Primary Children's Hospital	Salt Lake City	Utah
University of Virginia Division of Pediatric Cardiology	Charlottesville	Virginia
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
Hospital For Sick Children	Toronto	Ontario
Beijing Anzhen Hospital	Beijing	China
Guangzhou Women And Children's Medical Center	Guangzhou	China
Qingdao Women and Children's Hospital 1	Qingdao	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

+ 76 more sites — see the full list on the official registry below.

More Actelion trials in Germany

Outcome Study Assessing a 75 Milligrams (mg) Dose of Macitentan in Patients With Pulmonary Arterial Hypertension Active, not recruiting

A Clinical Study of to Confirm the Doses of Selexipag in Children With Pulmonary Arterial Hypertension Active, not recruiting

Other trials for the same condition

Riociguat in Children With Pulmonary Arterial Hypertension (PAH) Active, not recruiting

Endtidal Carbon Dioxide for Earlier Detection of Pulmonary Hypertension Enrolling by invitation

Extension Study of Sotatercept in People With Pulmonary Hypertension (MK-7962-023) Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04175600 on ClinicalTrials.gov ↗ ← All trials in Germany