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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT03775421
Registration number
NCT03775421
Ethics application status
Date submitted
21/11/2018
Date registered
14/12/2018
Titles & IDs
Public title
An Upcoming Clinical Study to Measure the Safety and Impact of a Drug Called Macitentan in Teenage and Adult Fontan Patients.
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Scientific title
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
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Secondary ID [1]
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2018-002821-45
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Secondary ID [2]
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AC-055H302
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Universal Trial Number (UTN)
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Trial acronym
RUBATO OL
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Congenital Heart Disease With Fontan Circulation
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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
Experimental: Open-label treatment period - oral administration of 10 mg macitentan once daily
Treatment: Drugs: macitentan 10 mg
macitentan 10 mg, 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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Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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Assessment method [1]
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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. Any AE occurring at or after the study treatment start up to 30 days after end of treatment (EOT) (limits included) within the analysis set was considered to be treatment-emergent.
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Timepoint [1]
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Up to 133 weeks
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Primary outcome [2]
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Number of Participants With Treatment-emergent Serious AEs (TESAEs)
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Assessment method [2]
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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. 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. Any SAE occurring at or after the study treatment start up to 30 days after EOT (limits included) within the analysis set was considered to be TESAEs.
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Timepoint [2]
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Up to 133 weeks
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Primary outcome [3]
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Number of Participants With TEAEs Leading to Death
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Assessment method [3]
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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. Any AE occurring at or after the study treatment start up to 30 days after EOT (limits included) within the analysis set was considered to be treatment-emergent.
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Timepoint [3]
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Up to 133 weeks
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Primary outcome [4]
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Number of Participants With TEAEs Leading to Premature Discontinuation of Study Treatment
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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. Any AE occurring at or after the study treatment start up to 30 days after EOT (limits included) within the analysis set was considered to be treatment-emergent.
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Timepoint [4]
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Up to 133 weeks
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Primary outcome [5]
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Number of Participants With Treatment-emergent Marked Laboratory Abnormalities up to 30 Days After Study Treatment Discontinuation
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Assessment method [5]
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Number of participants with treatment-emergent marked laboratory abnormalities (Hemoglobin \[gram/Liter {g/L}\], Platelets \[giga/L {10\^9 cells/L}\], Leukocytes \[10\^9 cells/L\], Lymphocytes \[10\^9 cells/L\], Neutrophils \[10\^9 cells/L\], Prothrombin International Normalized Ratio \[PINR;Ratio\], Aspartate Aminotransferase \[Units/L {U/L}\], Bilirubin \[micromoles/L {mcmol/L}\], Alkaline Phosphatase \[U/L\], Glomerular Filtration Rate \[milliliter/minute/1.73 meter square\], Glucose \[millimoles/L {mmol/L}\], Potassium \[mmol/L\], Sodium \[mmol/L\], Triglycerides \[mmol/L\] were reported. Abnormalities that occurred after study treatment start and up to 30 days after study treatment discontinuation, that were not present at baseline, were treatment-emergent. Marked laboratory abnormalities reported for at least 1 participant were reported in this outcome measure. \>=:greater than or equal to; \>:greater than; \<:less than; ULN: upper limit of normal; L:Low, H:High, LLL:lower/worse than LL, HHH:higher/worse than HH.
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Timepoint [5]
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Up to 133 weeks
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Primary outcome [6]
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Change From Baseline in Hemoglobin Over Time
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Assessment method [6]
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Change from baseline in hemoglobin over time was reported in this outcome measure.
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Timepoint [6]
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Baseline up to Week 130
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Primary outcome [7]
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Change From Baseline in Hematocrit Over Time
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Assessment method [7]
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Change from baseline in hematocrit over time was reported in this outcome measure.
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Timepoint [7]
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Baseline up to Week 130
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Primary outcome [8]
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Change From Baseline in Leukocytes, Neutrophils, Lymphocytes, and Platelets Over Time
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Assessment method [8]
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Change from baseline in leukocytes, neutrophils, lymphocytes, and platelets over time were reported in this outcome measure.
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Timepoint [8]
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Baseline up to Week 130
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Primary outcome [9]
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Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP) Over Time
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Assessment method [9]
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Change from baseline in systolic and diastolic arterial BP over time was reported in this outcome measure.
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Timepoint [9]
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Baseline up to Week 130
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Primary outcome [10]
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Change From Baseline in Pulse Rate Over Time
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Assessment method [10]
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Change from baseline in pulse rate over time was reported in this outcome measure.
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Timepoint [10]
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Baseline up to Week 130
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Primary outcome [11]
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Change From Baseline in Peripheral Oxygen Saturation (SpO2) Over Time
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Assessment method [11]
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Change from baseline in SpO2 over time was reported in this outcome measure.
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Timepoint [11]
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Baseline up to Week 130
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Primary outcome [12]
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Change From Baseline in Body Weight Over Time
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Assessment method [12]
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Change from baseline in body weight over time was reported in this outcome measure.
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Timepoint [12]
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Baseline up to Week 130
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Primary outcome [13]
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Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (AP), and Gamma Glutamyl Transferase (GGT) Over Time
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Assessment method [13]
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Change from baseline in ALT, AST, AP, and GGT over time were reported in this outcome measure.
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Timepoint [13]
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Baseline up to Week 130
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Primary outcome [14]
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Change From Baseline in Bilirubin, Direct Bilirubin, and Creatinine Over Time
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Assessment method [14]
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Change from baseline in bilirubin, direct bilirubin, and creatinine over time were reported in this outcome measure.
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Timepoint [14]
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Baseline up to Week 130
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Primary outcome [15]
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Change From Baseline in Glomerular Filtration Rate (GFR) Over Time
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Assessment method [15]
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Change from baseline in GFR over time was reported in this outcome measure.
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Timepoint [15]
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Baseline up to Week 130
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Primary outcome [16]
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Change From Baseline in Prothrombin Time Over Time
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Assessment method [16]
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Change from baseline in prothrombin time over time was reported in this outcome measure.
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Timepoint [16]
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Baseline up to Week 130
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Primary outcome [17]
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Change From Baseline in Prothrombin International Normalized Ratio Over Time
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Assessment method [17]
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Change from baseline in prothrombin international normalized ratio over time was reported in this outcome measure.
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Timepoint [17]
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Baseline up to Week 130
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Secondary outcome [1]
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Change From Baseline in Peak Oxygen Uptake/Consumption (VO2)
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Assessment method [1]
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Change from baseline in peak VO2 was reported in this outcome measure.
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Timepoint [1]
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Baseline, Week 52, and Week 104
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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)
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Assessment method [2]
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Change from baseline in mean count per minute of daily PA-Ac was reported in this outcome measure.
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Timepoint [2]
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Baseline, Week 26, Week 52, Week 78, and Week 104
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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.
* Subjects who have completed Week 52 of the parent AC-055H301/RUBATO DB study (NCT03153137)
* Women of childbearing potential must:
1. have a negative serum pregnancy test prior to first intake of OL study drug, and,
2. agree to perform monthly pregnancy tests up to the end of the safety follow up (S-FU) period, and,
3. use reliable methods of contraception from enrollment up to at least 30 days after study treatment discontinuation.
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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
* Clinical worsening leading to medical interventions including reoperation of Fontan circulation (Fontan take-down) during the enrollment period
* Systolic blood pressure < 90 mmHg (< 85 mmHg for subjects < 18 years old and < 150 cm of height) at rest
* Criteria related to macitentan use
* 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
NA
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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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
Stopped early
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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
11/04/2019
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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
18/01/2022
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Sample size
Target
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Accrual to date
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Final
112
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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 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 outside Australia
Country [1]
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United States of America
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State/province [1]
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Massachusetts
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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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Washington
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Country [3]
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Canada
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State/province [3]
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Quebec
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Country [4]
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China
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State/province [4]
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Beijing
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Country [5]
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China
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State/province [5]
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Shanghai
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Country [6]
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Czechia
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State/province [6]
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Praha 5
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Country [7]
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Denmark
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State/province [7]
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Copenhagen
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Country [8]
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France
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State/province [8]
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Paris
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Country [9]
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France
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State/province [9]
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Pessac
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Country [10]
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New Zealand
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State/province [10]
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Auckland
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Country [11]
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Poland
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State/province [11]
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Gdansk
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Country [12]
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Poland
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State/province [12]
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Krakow
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Country [13]
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Poland
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State/province [13]
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Wroclaw
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Country [14]
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Taiwan
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State/province [14]
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Taipei
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Country [15]
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United Kingdom
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State/province [15]
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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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Other collaborator category [1]
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Commercial sector/industry
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Name [1]
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Covance
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Address [1]
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Country [1]
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Other collaborator category [2]
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Other
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Name [2]
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Henry Ford Health System
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Address [2]
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Country [2]
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Other collaborator category [3]
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Other
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Name [3]
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Almac Clinical Technologies
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Address [3]
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Country [3]
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Other collaborator category [4]
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Other
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Name [4]
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ActiGraph LLC
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Address [4]
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Country [4]
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Other collaborator category [5]
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Commercial sector/industry
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Name [5]
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Medidata Solutions
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Address [5]
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Country [5]
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Ethics approval
Ethics application status
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Summary
Brief 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.
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Trial website
https://clinicaltrials.gov/study/NCT03775421
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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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Thierry Francis Briand, MD
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Address
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Actelion
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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
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
The data sharing policy of the Janssen Pharmaceutical Companies of Johnson \& Johnson is available at www.janssen.com/clinical-trials\\transparency. As noted on this site, requests for access to the study data can be submitted through Yale open Access (YODA) Project site at yoda.yale.edu
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.janssen.com/clinical-trials/transparency
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/21/NCT03775421/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/21/NCT03775421/SAP_002.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03775421