Clinical Trial: A Study to Find Out Whether the Medicine Macitentan Works in Children With Pulmonary Arterial Hypertension (PAH)

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: TOMORROW: pediaTric Use Of Macitentan tO Delay Disease pRogRessiOn in PAH Worldwide

Brief Summary: This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and PK of macitentan in children.

Detailed Summary:
Sponsor: Actelion

Current Primary Outcome: Time to the first CEC-confirmed disease progression event [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]

Time to the first of the following CEC-confirmed disease progression events: • Death (all causes) • Atrial septostomy or Potts' anastomosis, or registration on lung transplant list • Hospitalization due to worsening PAH • Clinical worsening of PAH


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Time to first CEC-confirmed hospitalization for PAH occurring between randomization and EOS [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
    Time to first CEC-confirmed hospitalization for PAH occurring between randomization and EOS
  • Time to CEC-confirmed death due to PAH occurring between randomization and EOS [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
    Time to CEC-confirmed death due to PAH occurring between randomization and EOS
  • Time to death (all causes) occurring between randomization and Study Closure [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
    Time to death (all causes) occurring between randomization and Study Closure


Original Secondary Outcome:

  • Time to first CEC-confirmed hospitalization for PAH occurring between randomization and EOS [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
  • Time to CEC-confirmed death due to PAH occurring between randomization and EOS [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]
  • Time to death (all causes) occurring between randomization and Study Closure [ Time Frame: Between randomization and EOS/study closure; up to 6 years ]


Information By: Actelion

Dates:
Date Received: October 12, 2016
Date Started: July 30, 2017
Date Completion: July 1, 2022
Last Updated: April 25, 2017
Last Verified: April 2017