NCT02932410

A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)

A Multicenter, Open-label, Randomized, Study With Single-arm Extension Period to Assess the Pharmacokinetics, Safety and Efficacy of Macitentan Versus Standard of Care in Children With Pulmonary Arterial Hypertension

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 165 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 1 Month – 17 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with pulmonary arterial hypertension (PAH).

Conditions

• Pulmonary Arterial Hypertension

Interventions

Type	Name	Description
DRUG	Macitentan	Dispersible tablet; Oral use
OTHER	Standard-of-care	Standard-of-care as per site's clinical practice which may comprise treatment with PAH non-specific treatment and/or up to two PAH-specific medications excluding macitentan and IV/SC prostanoids.

Timeline

• Start date: 2017-10-24
• Primary completion: 2024-08-28
• Completion: 2025-11-27
• First posted: 2016-10-13
• Last updated: 2026-03-13
• Results posted: 2025-09-12

Locations

85 sites across 23 countries: United States, Australia, Austria, Brazil, Canada, China, Colombia, Finland, France, Hungary, Israel, Malaysia, Mexico, Philippines, Poland, Portugal, Russia, South Africa, South Korea, Spain, Thailand, Ukraine, Vietnam

Regulatory

• FDA-regulated drug study