Efficacy and safety of macitentan in Fontan-palliated patients: 52-week randomized, placebo-controlled RUBATO phase 3 trial and open-label extension.
Clift, Paul Berger, Felix Sondergaard, Lars Antonova, Petra Disney, Patrick Nicolarsen, Jeremy Thambo, Jean-Benoît Pajak, Lidia Tomkiewicz Wang, Jou-Kou Jensen, Annette Schophuus
Clift, Paul
Berger, Felix
Sondergaard, Lars
Antonova, Petra
Disney, Patrick
Nicolarsen, Jeremy
Thambo, Jean-Benoît
Pajak, Lidia Tomkiewicz
Wang, Jou-Kou
Jensen, Annette Schophuus
Abstract
Patients aged ≥12 years, New York Heart Association functional class II/III, underwent total cavopulmonary connection >1 year pre-screening and showed no signs of Fontan failure/clinical deterioration. In RUBATO-DB, primary efficacy endpoint was change in peak oxygen consumption (VO2) from baseline to week 16; secondary endpoints were change from baseline over 52 weeks in peak VO2 and change in mean count/minute of daily physical activity via accelerometer (PA-Ac) from baseline to week 16. Safety was assessed throughout both studies.
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Date
2024-08-29
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Clift P, Berger F, Sondergaard L, Antonova P, Disney P, Nicolarsen J, Thambo JB, Tomkiewicz Pajak L, Wang JK, Schophuus Jensen A, Efficace M, Friberg M, Haberle D, Walter V, d'Udekem Y. Efficacy and safety of macitentan in Fontan-palliated patients: 52-week randomized, placebo-controlled RUBATO Phase 3 trial and open-label extension. J Thorac Cardiovasc Surg. 2025 Feb;169(2):385-394.e5. doi: 10.1016/j.jtcvs.2024.08.039. Epub 2024 Aug 29