NCT04175600

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

A Randomized, Multicenter, Double-Blind, Placebo-Controlled, Parallel-Group Study With Open-Label Extension Period to Assess the Efficacy and Safety of Selexipag as Add-On Treatment to Standard of Care in Children Aged >=2 to <18 Years With Pulmonary Arterial Hypertension

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 138 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	Selexipag	Selexipag tablet will be administered orally.
DRUG	Placebo	Matching placebo tablets will be administered orally.
DRUG	Standard of Care (SOC): Endothelin receptor antagonist	ERAs will be administered as SOC therapy.
DRUG	SOC: Phosphodiesterase type 5 (PDE-5) inhibitor	PDE-5 inhibitor will be administered as SOC therapy.
DRUG	SOC: Soluble guanylate cyclase stimulator	Soluble guanylate cyclase stimulator will be administered as SOC therapy.

Timeline

• Start date: 2020-01-16
• Primary completion: 2024-10-11
• Completion: 2027-10-01
• First posted: 2019-11-25
• Last updated: 2026-04-13

Locations

116 sites across 32 countries: 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

Regulatory

• FDA-regulated drug study