Stopped: As The RUBATO DB study (NCT03153137) did not show any benefit of treatment with macitentan in Fontan-palliated participants, the sponsor has decided to terminate the RUBATO OL study (NCT03775421). No new safety observations were made.
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.
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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Timeframe: Up to 133 weeks
Number of Participants With Treatment-emergent Serious AEs (TESAEs)
Timeframe: Up to 133 weeks
Number of Participants With TEAEs Leading to Death
Timeframe: Up to 133 weeks
Number of Participants With TEAEs Leading to Premature Discontinuation of Study Treatment
Timeframe: Up to 133 weeks
Number of Participants With Treatment-emergent Marked Laboratory Abnormalities up to 30 Days After Study Treatment Discontinuation
Timeframe: Up to 133 weeks
Change From Baseline in Hemoglobin Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Hematocrit Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Leukocytes, Neutrophils, Lymphocytes, and Platelets Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP) Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Pulse Rate Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Peripheral Oxygen Saturation (SpO2) Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Body Weight Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (AP), and Gamma Glutamyl Transferase (GGT) Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Bilirubin, Direct Bilirubin, and Creatinine Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Glomerular Filtration Rate (GFR) Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Prothrombin Time Over Time
Timeframe: Baseline up to Week 130
Change From Baseline in Prothrombin International Normalized Ratio Over Time
Timeframe: Baseline up to Week 130