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

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).

Timeline

Start

2017-10-24

Primary completion

2024-08-28

Completion

2025-11-27

Publications

• Berger RMF, Dunbar Ivy D, Borissoff JI, Cerovic S, Csonka D, Remenova T, Richard D, Villeneuve M, Beghetti M. Macitentan in Pediatric Pulmonary Arterial Hypertension (TOMORROW): A Randomized Clinical Trial. J Pediatr. 2026 Jun;293:115057. doi: 10.1016/j.jpeds.2026.115057. Epub 2026 Mar 6.

Drugs

Indications