NCT03775421

An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients.

Prospective, Multi-center, Single-arm, Open-label Long-term Study Assessing the Safety, Tolerability, and Effectiveness of Macitentan in Fontan-palliated Adult and Adolescent Subjects

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 112 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The aim of this open-label (OL) trial is to study the long-term use of macitentan for up to 2 years in Fontan-palliated adult and adolescent patients beyond the 52 weeks of treatment in the parent RUBATO double-blind (DB) study (AC-055H301, NCT03153137). This OL trial studies the long-term effect of macitentan in Fontan-palliated patients as it is not known if the effect of macitentan is sustained beyond 52 weeks (end of the parent RUBATO DB study). In addition, the trial also studies the long-term safety of macitentan as this is also unknown. Furthermore, the opportunity will be given to patients who were on placebo in the parent RUBATO DB study to receive macitentan 10 mg and benefit from a potentially active treatment.

Conditions

• Congenital Heart Disease With Fontan Circulation

Interventions

Type	Name	Description
DRUG	macitentan 10 mg	macitentan 10 mg, film-coated tablet, oral use

Timeline

• Start date: 2019-04-11
• Primary completion: 2022-01-18
• Completion: 2022-01-18
• First posted: 2018-12-14
• Last updated: 2023-03-07
• Results posted: 2023-03-07

Locations

19 sites across 11 countries: United States, Australia, Canada, China, Czechia, Denmark, France, New Zealand, Poland, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study