Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT03775421
Registration number
NCT03775421
Ethics application status
Date submitted
21/11/2018
Date registered
14/12/2018
Date last updated
7/03/2023
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.
Query!
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
Query!
Secondary ID [1]
0
0
2018-002821-45
Query!
Secondary ID [2]
0
0
AC-055H302
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
RUBATO OL
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Congenital Heart Disease With Fontan Circulation
0
0
Query!
Condition category
Condition code
Cardiovascular
0
0
0
0
Query!
Coronary heart disease
Query!
Cardiovascular
0
0
0
0
Query!
Other cardiovascular diseases
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
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
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Up to 133 weeks
Query!
Primary outcome [2]
0
0
Number of Participants With Treatment-emergent Serious AEs (TESAEs)
Query!
Assessment method [2]
0
0
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.
Query!
Timepoint [2]
0
0
Up to 133 weeks
Query!
Primary outcome [3]
0
0
Number of Participants With TEAEs Leading to Death
Query!
Assessment method [3]
0
0
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.
Query!
Timepoint [3]
0
0
Up to 133 weeks
Query!
Primary outcome [4]
0
0
Number of Participants With TEAEs Leading to Premature Discontinuation of Study Treatment
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Up to 133 weeks
Query!
Primary outcome [5]
0
0
Number of Participants With Treatment-emergent Marked Laboratory Abnormalities up to 30 Days After Study Treatment Discontinuation
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Up to 133 weeks
Query!
Primary outcome [6]
0
0
Change From Baseline in Hemoglobin Over Time
Query!
Assessment method [6]
0
0
Change from baseline in hemoglobin over time was reported in this outcome measure.
Query!
Timepoint [6]
0
0
Baseline up to Week 130
Query!
Primary outcome [7]
0
0
Change From Baseline in Hematocrit Over Time
Query!
Assessment method [7]
0
0
Change from baseline in hematocrit over time was reported in this outcome measure.
Query!
Timepoint [7]
0
0
Baseline up to Week 130
Query!
Primary outcome [8]
0
0
Change From Baseline in Leukocytes, Neutrophils, Lymphocytes, and Platelets Over Time
Query!
Assessment method [8]
0
0
Change from baseline in leukocytes, neutrophils, lymphocytes, and platelets over time were reported in this outcome measure.
Query!
Timepoint [8]
0
0
Baseline up to Week 130
Query!
Primary outcome [9]
0
0
Change From Baseline in Systolic and Diastolic Arterial Blood Pressure (BP) Over Time
Query!
Assessment method [9]
0
0
Change from baseline in systolic and diastolic arterial BP over time was reported in this outcome measure.
Query!
Timepoint [9]
0
0
Baseline up to Week 130
Query!
Primary outcome [10]
0
0
Change From Baseline in Pulse Rate Over Time
Query!
Assessment method [10]
0
0
Change from baseline in pulse rate over time was reported in this outcome measure.
Query!
Timepoint [10]
0
0
Baseline up to Week 130
Query!
Primary outcome [11]
0
0
Change From Baseline in Peripheral Oxygen Saturation (SpO2) Over Time
Query!
Assessment method [11]
0
0
Change from baseline in SpO2 over time was reported in this outcome measure.
Query!
Timepoint [11]
0
0
Baseline up to Week 130
Query!
Primary outcome [12]
0
0
Change From Baseline in Body Weight Over Time
Query!
Assessment method [12]
0
0
Change from baseline in body weight over time was reported in this outcome measure.
Query!
Timepoint [12]
0
0
Baseline up to Week 130
Query!
Primary outcome [13]
0
0
Change From Baseline in Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Alkaline Phosphatase (AP), and Gamma Glutamyl Transferase (GGT) Over Time
Query!
Assessment method [13]
0
0
Change from baseline in ALT, AST, AP, and GGT over time were reported in this outcome measure.
Query!
Timepoint [13]
0
0
Baseline up to Week 130
Query!
Primary outcome [14]
0
0
Change From Baseline in Bilirubin, Direct Bilirubin, and Creatinine Over Time
Query!
Assessment method [14]
0
0
Change from baseline in bilirubin, direct bilirubin, and creatinine over time were reported in this outcome measure.
Query!
Timepoint [14]
0
0
Baseline up to Week 130
Query!
Primary outcome [15]
0
0
Change From Baseline in Glomerular Filtration Rate (GFR) Over Time
Query!
Assessment method [15]
0
0
Change from baseline in GFR over time was reported in this outcome measure.
Query!
Timepoint [15]
0
0
Baseline up to Week 130
Query!
Primary outcome [16]
0
0
Change From Baseline in Prothrombin Time Over Time
Query!
Assessment method [16]
0
0
Change from baseline in prothrombin time over time was reported in this outcome measure.
Query!
Timepoint [16]
0
0
Baseline up to Week 130
Query!
Primary outcome [17]
0
0
Change From Baseline in Prothrombin International Normalized Ratio Over Time
Query!
Assessment method [17]
0
0
Change from baseline in prothrombin international normalized ratio over time was reported in this outcome measure.
Query!
Timepoint [17]
0
0
Baseline up to Week 130
Query!
Secondary outcome [1]
0
0
Change From Baseline in Peak Oxygen Uptake/Consumption (VO2)
Query!
Assessment method [1]
0
0
Change from baseline in peak VO2 was reported in this outcome measure.
Query!
Timepoint [1]
0
0
Baseline, Week 52, and Week 104
Query!
Secondary outcome [2]
0
0
Change From Baseline in Mean Count Per Minute of Daily Physical Activity Measured by Accelerometer (PA-Ac)
Query!
Assessment method [2]
0
0
Change from baseline in mean count per minute of daily PA-Ac was reported in this outcome measure.
Query!
Timepoint [2]
0
0
Baseline, Week 26, Week 52, Week 78, and Week 104
Query!
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.
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
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
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
N/A
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Terminated
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
11/04/2019
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
18/01/2022
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
112
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Royal Adelaide Hospital - Adelaide
Query!
Recruitment hospital [2]
0
0
Royal Prince Alfred Hospital - Camperdown
Query!
Recruitment hospital [3]
0
0
The Prince Charles Hospital, Adult Congenital Heart Disease Unit - Chermside
Query!
Recruitment hospital [4]
0
0
Royal Children's Hospital - Parkville
Query!
Recruitment postcode(s) [1]
0
0
5000 - Adelaide
Query!
Recruitment postcode(s) [2]
0
0
2050 - Camperdown
Query!
Recruitment postcode(s) [3]
0
0
4032 - Chermside
Query!
Recruitment postcode(s) [4]
0
0
3052 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Massachusetts
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Washington
Query!
Country [3]
0
0
Canada
Query!
State/province [3]
0
0
Quebec
Query!
Country [4]
0
0
China
Query!
State/province [4]
0
0
Beijing
Query!
Country [5]
0
0
China
Query!
State/province [5]
0
0
Shanghai
Query!
Country [6]
0
0
Czechia
Query!
State/province [6]
0
0
Praha 5
Query!
Country [7]
0
0
Denmark
Query!
State/province [7]
0
0
Copenhagen
Query!
Country [8]
0
0
France
Query!
State/province [8]
0
0
Paris
Query!
Country [9]
0
0
France
Query!
State/province [9]
0
0
Pessac
Query!
Country [10]
0
0
New Zealand
Query!
State/province [10]
0
0
Auckland
Query!
Country [11]
0
0
Poland
Query!
State/province [11]
0
0
Gdansk
Query!
Country [12]
0
0
Poland
Query!
State/province [12]
0
0
Krakow
Query!
Country [13]
0
0
Poland
Query!
State/province [13]
0
0
Wroclaw
Query!
Country [14]
0
0
Taiwan
Query!
State/province [14]
0
0
Taipei
Query!
Country [15]
0
0
United Kingdom
Query!
State/province [15]
0
0
Birmingham
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Actelion
Query!
Address
Query!
Country
Query!
Other collaborator category [1]
0
0
Commercial sector/Industry
Query!
Name [1]
0
0
Covance
Query!
Address [1]
0
0
Query!
Country [1]
0
0
Query!
Other collaborator category [2]
0
0
Other
Query!
Name [2]
0
0
Henry Ford Health System
Query!
Address [2]
0
0
Query!
Country [2]
0
0
Query!
Other collaborator category [3]
0
0
Other
Query!
Name [3]
0
0
Almac Clinical Technologies
Query!
Address [3]
0
0
Query!
Country [3]
0
0
Query!
Other collaborator category [4]
0
0
Other
Query!
Name [4]
0
0
ActiGraph LLC
Query!
Address [4]
0
0
Query!
Country [4]
0
0
Query!
Other collaborator category [5]
0
0
Commercial sector/Industry
Query!
Name [5]
0
0
Medidata Solutions
Query!
Address [5]
0
0
Query!
Country [5]
0
0
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT03775421
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Thierry Francis Briand, MD
Query!
Address
0
0
Actelion
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03775421
Download to PDF