Clinical Trials in France / NCT04175600
Active, not recruiting
Phase 3
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 France tracker
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
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 | Status |
|---|---|---|---|
| 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 Childrens 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 France
Other trials for the same condition
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 France