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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03153137
Registration number
NCT03153137
Ethics application status
Date submitted
12/05/2017
Date registered
15/05/2017
Date last updated
13/09/2022
Titles & IDs
Public title
Clinical Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Subjects
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Scientific title
Prospective, Multi-center, Double-blind, Randomized, Placebo-controlled, Parallel-group Study Assessing the Efficacy and Safety of Macitentan in Fontan-palliated Adult and Adolescent Subjects
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Secondary ID [1]
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2016-003320-23
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Secondary ID [2]
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AC-055H301
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Universal Trial Number (UTN)
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Trial acronym
RUBATO
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Congenital Heart Disease
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Condition category
Condition code
Cardiovascular
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Coronary heart disease
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Cardiovascular
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Other cardiovascular diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Macitentan 10 mg
Treatment: Drugs - Placebo
Experimental: Macitentan - Macitentan 10 mg per day; film-coated tablet; oral use
Placebo Comparator: Placebo - film-coated tablet; oral use
Treatment: Drugs: Macitentan 10 mg
film-coated tablet; oral use
Treatment: Drugs: Placebo
film-coated tablet; oral use
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change From Baseline in Peak Oxygen Uptake/Consumption (VO2) Up to Week 16
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Assessment method [1]
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Change from baseline in peak VO2 up to Week 16 was reported.
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Timepoint [1]
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Baseline up to Week 16
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Secondary outcome [1]
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Change From Baseline in Peak VO2 Up to Week 52
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Assessment method [1]
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Change from baseline in peak VO2 up to Week 52 was reported.
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Timepoint [1]
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Baseline up to Week 52
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Secondary outcome [2]
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Change From Baseline in Mean Count Per Minute of Daily Physical Activity Measured by Accelerometer (PA-Ac) Up to Week 16
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Assessment method [2]
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Change from baseline in mean count per minute of daily PA-Ac up to Week 16 was reported. The daily physical activity (counts per min) of the participant was assessed via accelerometer during daytime. The accelerometer was given to the participant at Visit 1, and data was collected for 9 consecutive daily daytime periods after Visit 1 (baseline) to Visit 4 (Week 16).
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Timepoint [2]
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Baseline up to Week 16
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Secondary outcome [3]
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Number of Participants With Treatment-emergent Serious Adverse Events (SAEs)
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Assessment method [3]
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SAE is any AE that results in: death, persistent or significant disability/incapacity, requires inpatient hospitalization or prolongation of existing hospitalization, is life-threatening experience, is a congenital anomaly/birth defect and may jeopardize participant and/or may require medical or surgical intervention to prevent one of the outcomes listed above.
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Timepoint [3]
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Up to 56 weeks
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Secondary outcome [4]
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Number of Participants With Treatment-emergent Adverse Events (AEs)
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Assessment method [4]
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An adverse event is any untoward medical event that occurs in a participant administered an investigational product, and it does not necessarily indicate only events with clear causal relationship with the relevant investigational product.
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Timepoint [4]
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Up to 56 weeks
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Secondary outcome [5]
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Number of Participants With AEs Leading to Premature Discontinuation of Study Treatment
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Assessment method [5]
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Number of participants with AEs leading to premature discontinuation of study treatment was reported. AEs leading to premature discontinuation of study treatment were those with action taken with study drug reported as 'permanently discontinued' by the investigator.
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Timepoint [5]
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Up to 56 weeks
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Secondary outcome [6]
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Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP)
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Assessment method [6]
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Change from baseline in systolic and diastolic arterial BP at Week 8, Week 16, Week 32 and Week 52 was reported.
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Timepoint [6]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [7]
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Change From Baseline in Pulse Rate
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Assessment method [7]
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Change from baseline in pulse rate at Week 8, Week 16, Week 32 and Week 52 was reported.
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Timepoint [7]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [8]
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Change From Baseline in Oxygen Saturation (SpO2)
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Assessment method [8]
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Change from baseline in SpO2 was reported.
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Timepoint [8]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [9]
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Change From Baseline in Body Weight
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Assessment method [9]
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Change from baseline in body weight was reported.
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Timepoint [9]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [10]
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Number of Participants With Treatment-emergent Markedly Abnormal Laboratory Values
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Assessment method [10]
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Number of participants with treatment-emergent markedly laboratory abnormal laboratory values were reported. Abnormal values for platelets (LL < 75); Lymphocytes (HH > 4.0); Neutrophils (LL < 1.5); Prothrombin International Normalized Ratio: HH (greater than and equal to [>=] 1.5 upper limit of normal [ULN]), Ratio: HH >= 2.5 ULN); Bilirubin (HH >= 2 ULN); Alkaline Phosphatase (HH > 2.5 ULN); Glomerular Filtration Rate (LL < 60); Glucose (HH > 8.9); Triglycerides (HH > 3.42). Here "HH" refers to values above the normal range, where H stands for "high" and "LL" refers to values below the normal range where L stands for "low".
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Timepoint [10]
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Up to 56 weeks
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Secondary outcome [11]
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Change From Baseline in Hemoglobin
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Assessment method [11]
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Change from baseline in hemoglobin was reported.
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Timepoint [11]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [12]
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Change From Baseline in Hematocrit
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Assessment method [12]
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Change from baseline in hematocrit was reported.
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Timepoint [12]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [13]
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Change From Baseline in Erythrocytes and Reticulocytes
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Assessment method [13]
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Change from baseline in erythrocytes and reticulocytes at Week 8, Week 16, Week 32 and Week 52 was reported.
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Timepoint [13]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [14]
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Change From Baseline in Leucocytes, Neutrophils, Lymphocytes, Monocytes, Eosinophils, Basophils and Platelets
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Assessment method [14]
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Change from baseline in leucocytes, neutrophils, lymphocytes, monocytes, eosinophils, basophils and platelets at Week 8, Week 16, Week 32 and Week 52 was reported.
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Timepoint [14]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [15]
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Change From Baseline in Prothrombin Time
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Assessment method [15]
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Change from baseline in prothrombin time was reported.
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Timepoint [15]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [16]
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Change From Baseline in Prothrombin International Normalized Ratio
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Assessment method [16]
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Change from baseline in prothrombin international normalized ratio was reported.
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Timepoint [16]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [17]
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Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) and Alkaline Phosphatase (AP)
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Assessment method [17]
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Change from baseline in ALT, AST and AP were reported.
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Timepoint [17]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [18]
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Change From Baseline in Bilirubin and Direct Bilirubin
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Assessment method [18]
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Change from baseline in bilirubin and direct bilirubin was reported.
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Timepoint [18]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [19]
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Change From Baseline in Gamma Glutamyl Transferase
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Assessment method [19]
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Change from baseline in gamma glutamyl transferase was reported.
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Timepoint [19]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [20]
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Change From Baseline in Creatinine
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Assessment method [20]
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Change from baseline in creatinine was reported.
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Timepoint [20]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [21]
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Change From Baseline in Urea Nitrogen
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Assessment method [21]
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Change from baseline in urea nitrogen was reported.
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Timepoint [21]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [22]
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Change From Baseline in Urate
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Assessment method [22]
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Change from baseline in urate was reported.
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Timepoint [22]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [23]
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Change From Baseline in Glucose, Cholesterol, Triglycerides, Sodium, Potassium, Chloride and Calcium
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Assessment method [23]
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Change from baseline in glucose, cholesterol, triglycerides, sodium, potassium, chloride and calcium was reported.
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Timepoint [23]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [24]
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Change From Baseline in Albumin and Protein
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Assessment method [24]
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Change from baseline in albumin and protein was reported.
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Timepoint [24]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [25]
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Change From Baseline in Alpha Fetoprotein
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Assessment method [25]
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Change from baseline in alpha fetoprotein was reported.
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Timepoint [25]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Secondary outcome [26]
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Change From Baseline in Cystatin C
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Assessment method [26]
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Change from baseline in cystatin C was reported.
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Timepoint [26]
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Baseline, Week 8, Week 16, Week 32 and Week 52
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Eligibility
Key inclusion criteria
- Written informed consent/assent from the subject and/or a legal representative prior
to initiation of any study-mandated procedures
- Fontan-palliated subjects with either intra-atrial lateral tunnel total cavopulmonary
connection (LT-TCPC), or extra cardiac tunnel TCPC (EC-TCPC) surgery > 1 year before
Screening. Either LT- or EC-TCPC can be primary or secondary to atrio-pulmonary
connection
- New York Heart Association (NYHA) functional class (FC) II or III (assessed by the
investigator using the Specific Activity Scale
- Women of childbearing potential must have a negative serum pregnancy test use reliable
contraception
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Pattern of Fontan circulation severity
- Deterioration of the Fontan-palliated condition.
- Limitations to Cardiopulmonary exercise testing (CPET)
- Peak VO2 < 15 mL/kg/min.
- Any known factor or disease that may interfere with treatment compliance or full
participation in the study
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
14/08/2017
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
26/07/2021
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Sample size
Target
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Accrual to date
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Final
142
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Royal Adelaide Hospital - Adelaide
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Recruitment hospital [2]
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Royal Prince Alfred Hospital - Camperdown
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Recruitment hospital [3]
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The Prince Charles Hospital, Adult Congenital Heart Disease Unit - Chermside
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Recruitment hospital [4]
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Royal Children's Hospital - Parkville
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Recruitment hospital [5]
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Queensland CHILDREN'S HOSPITAL - South Brisbane
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Recruitment hospital [6]
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Westmead Hospital - Westmead
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Recruitment postcode(s) [1]
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5000 - Adelaide
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Recruitment postcode(s) [2]
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2050 - Camperdown
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Recruitment postcode(s) [3]
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4032 - Chermside
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Recruitment postcode(s) [4]
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3052 - Parkville
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Recruitment postcode(s) [5]
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4101 - South Brisbane
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Recruitment postcode(s) [6]
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2145 - Westmead
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Massachusetts
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Country [4]
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United States of America
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State/province [4]
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Ohio
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Country [5]
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United States of America
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State/province [5]
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Texas
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Country [6]
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United States of America
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State/province [6]
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Washington
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Country [7]
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Canada
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State/province [7]
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Quebec
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Country [8]
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China
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State/province [8]
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Beijing
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Country [9]
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China
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State/province [9]
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Shanghai
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Country [10]
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China
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State/province [10]
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Wuhan
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Country [11]
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Czechia
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State/province [11]
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Praha 5
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Country [12]
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Denmark
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State/province [12]
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Copenhagen
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Country [13]
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France
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State/province [13]
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Montpellier Cedex 5
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Country [14]
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France
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State/province [14]
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Paris
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Country [15]
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France
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State/province [15]
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Pessac
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Country [16]
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Germany
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State/province [16]
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Berlin
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Country [17]
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Germany
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State/province [17]
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München
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Country [18]
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New Zealand
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State/province [18]
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Auckland
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Country [19]
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Poland
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State/province [19]
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Gdansk
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Country [20]
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Poland
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State/province [20]
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Krakow
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Country [21]
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Poland
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State/province [21]
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Kraków
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Country [22]
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Poland
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State/province [22]
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Wroclaw
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Country [23]
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Taiwan
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State/province [23]
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Taipei
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Country [24]
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United Kingdom
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State/province [24]
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Birmingham
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Actelion
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective is to assess the effect of macitentan 10 mg as compared to placebo on
exercise capacity through cardiopulmonary exercise testing.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03153137
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03153137
Download to PDF